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Corporate Partner news

This section of the WFH website is for manufacturers to post press releases of interest to the global bleeding disorders community.

Information posted here does not represent an endorsement of a particular product by the WFH. Statements and opinions expressed here do not necessarily represent the opinions, policies, or recommendations of the WFH.

Spark Therapeutics, a member of the Roche Group, introduces a new FVIII gene therapy variant while discontinuing the phase 3 dirloctocogene samoparvovec gene therapy study
December 6, 2024 – Spark Therapeutics has announced the decision to discontinue the phase 3 study of dirloctocogene samoparvovec gene therapy, while introducing an enhanced-function Factor VIII variant, aiming to develop a durable gene therapy product. Building on the capsid technology and scientific insights from the dirloctocogene samoparvovec program, Spark plans to initiate a phase 2b clinical study of the enhanced-function variant in the second half of 2025. The phase 3 dirloctocogene samoparvovec study was stopped before any participants were dosed, and the decision was not related to safety concerns with dirloctocogene samoparvovec.


European Commission approves marstacimab treatment for hemophilia A or B
November 20, 2024 — The European Commissionhas approved marstacimab—developed by Pfizer—for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and adolescent patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies). A notable benefit of marstacimab is that it is delivered subcutaneously once weekly through a pre-filled pen or syringe. LINK


Centessa Pharmaceuticals announces discontinuation of SerpinPC development
November 12, 2024 — Centessa Pharmaceuticals will discontinue the global clinical development of SerpinPC, an investigational serine protease inhibitor (SERPIN) engineered to specifically inhibit Activated Protein C (APC). SerpinPC was observed to have a favourable safety and tolerability profile; however, considering the evolving treatment and market landscape for hemophilia B, including the recent FDA approval of a similar product, Centessa decided to stop any further investment in this program. LINK


Spark Therapeutics pauses Phase 3 dirloctocogene samoparvovec gene therapy clinical study
October 16, 2024 — The following announcement was received by Spark:
“After careful consideration, Spark Therapeutics, a member of the Roche Group, has elected to pause the Phase 3 dirloctocogene samoparvovec gene therapy clinical study while we evaluate the possibility to further transform outcomes for hemophilia A patients with an adeno-associated virus (AAV) gene therapy. This pause is not related to safety concerns with dirloctocogene samoparvovec. Spark will continue to work with investigators to ensure the necessary long-term follow-up with patients who have received dirloctocogene samoparvovec. Spark is dedicated to developing gene therapies that demonstrate safety, efficacy, durability, and predictability. Spark will provide an update as soon as they are able.”


FDA approves marstacimab-hncq treatment for hemophilia A or B
October 11, 2024 — The U.S. Food and Drug Administration (FDA) has approved marstacimab-hncq—developed by Pfizer—for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies). A notable benefit of marstacimab-hncq is that it is delivered subcutaneously once weekly through a pre-filled pen or syringe. LINK


Fidanacogene elaparvovec gene therapy approved for Europe
July 25, 2024 — The European Commission has approved fidanacogene elaparvovec, an adeno-associated viral (AAV) vector-based gene therapy for the treatment of hemophilia B in Europe. Developed by Pfizer, the treatment is for adults (aged 18 years or older) with moderately severe to severe hemophilia B (congenital Factor IX deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74. LINK


NEJM publishes phase 3 data supporting the use of efanesoctocog alfa for treating children with severe hemophilia A
July 18, 2024 — Full results from the XTEND-Kids phase 3 study published in The New England Journal of Medicine (NEJM) highlights the efficacy, safety, and pharmacokinetic profile of Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein. Efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy, is approved for adults and children with hemophilia A for routine prophylaxis and on-demand treatment to control bleeding episodes as well as for perioperative management (surgery). LINK


U.S. FDA approves gene therapy for hemophilia B
April 26, 2024 — Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved fidanacogene elaparvovec-dzkt for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:

  • Currently use factor IX prophylaxis therapy, or
  • Have current or historical life-threatening hemorrhage, or
  • Have repeated, serious spontaneous bleeding episodes, and,
  • Do not have neutralizing antibodies to the adeno-associated virus serotype used

LINK


Fidanacogene elaparvovec gene therapy approved for the treatment of hemophilia B

January 3, 2024 — Health Canada has approved fidanacogene elaparvovec, an adeno-associated viral (AAV) vector-based gene therapy for the treatment of hemophilia B which has been developed by Pfizer. The treatment is for adults (aged 18 years or older) with moderately severe to severe hemophilia B (congenital Factor IX deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74. LINK

BioMarin Announces First Person Treated Commercially with valoctocogene roxaparvovec-rvox
August 30, 2023 — BioMarin Pharmaceutical Inc. announced today that an individual in Germany with severe hemophilia A was treated with valoctocogene roxaparvovec-rvox, marking the first time that this gene therapy has been given commercially in Europe. LINK

Roche discontinues hemophilia A gene therapy treatment candidate
July 28, 2023 — Roche is discontinuing the development of a mid-stage gene therapy candidate which was previously being assessed as a treatment for hemophilia A patients with inhibitors to factor VIII, the company announced Thursday. LINK

U.S. Food and Drug Administration Approves BioMarin’s valoctocogene roxaparvovec-rvox, the first gene therapy for adults with severe hemophilia A
June 29, 2023 — BioMarin today announced that the United States Food and Drug Administration (FDA) approved valoctocogene roxaparvovec-rvox gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. LINK

CSL Behring Announces the first patient has received FDA-Approved etranacogene dezaparvovec-drlb for hemophilia B
June 20, 2023 — Etranacogene dezaparvovec-drlb (Hemgenix), the first FDA approved gene therapy for hemophilia B, has now been administered for the first time as an approved product to a person with hemophilia B. Hemgenix is also approved for use in the EU and has been granted conditional marketing authorization by the UK’s Medicines and Healthcare product Regulatory Agency. LINK

Two fitusiran Phase 3 studies published in The Lancet and The Lancet Haematology highlight potential to address unmet needs across all types of hemophilia.
April 4, 2023 — Both Phase 3 studies achieved their primary and secondary endpoints; fitusiran prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection in people with hemophilia with and without inhibitors. LINK

BioMarin provides update on FDA review of valoctocogene roxaparvovec gene therapy for adults with severe hemophilia A
March 6, 2023 — BioMarin Pharmaceutical Inc. announced today that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for adults with severe hemophilia A. The FDA determined that the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study constituted a Major Amendment due to the substantial amount of additional data and set a new PDUFA Target Action Date of June 30, 2023. The company had previously communicated that this data submission could be qualified as a Major Amendment. LINK

Completed XTEND-Kids Phase 3 Sanofi and Sobi study strengthens potential of Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl for treatment of children <12 years of age with hemophilia A
March 2, 2023 — The XTEND-Kids phase 3 Sanofi and Sobi study evaluating the safety, efficacy and pharmacokinetics of Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl as once-weekly prophylaxis in previously treated patients <12 years of age with severe hemophilia A met its primary endpoint of safety, with no FVIII inhibitors detected in 74 children, with more than 50 children experiencing at least 50 exposure days, nearly a full year of treatment. The completion of XTEND-Kids represents the final milestone needed for regulatory submission in the EU. LINK

FDA approves once-weekly Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl a new class of factor VIII therapy for hemophilia A
February 23, 2023 – Sanofi announces that the U.S. Food and Drug Administration (FDA) has approved Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl, previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. This treatment is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. LINK.

NEJM publishes once-weekly efanesoctocog alfa Phase 3 data for people with hemophilia A
January 25, 2023 – Pivotal study data published in The New England Journal of Medicine (NEJM) highlights the efficacy, safety, and pharmacokinetic profile of efanesoctocog alfa, an investigational treatment for hemophilia A. These data demonstrate that efanesoctocog alfa delivered normal to near-normal factor activity levels (>40%) for the majority of the week with once-weekly dosing. LINK

BioMarin Announces Stable and Durable Annualized Bleed Control at 3 years for ROCTAVIAN™
January 8, 2023 — BioMarin Pharmaceutical Inc. today announced positive results from three years of follow up from its ongoing global Phase 3 GENEr8-1 study of ROCTAVIANTM(valoctocogene roxaparvovec), an investigational one-time gene therapy for the treatment of adults with severe hemophilia A. This is the largest and longest global Phase 3 study to date for any gene therapy in hemophilia with 134 participants. LINK

Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate
December 29, 2022 — Pfizer Inc. today announced positive top-line results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. LINK

EMA recommends granting a conditional marketing authorisation in EU for Hemgenix
December 16, 2022 — The European Medicines Agency (EMA) has recommended granting a conditional marketing authorisation in the European Union (EU) for CSL Behring GmbH’s Hemgenix (etranacogene dezaparvovec) for the treatment of severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system which make factor IX medicines less effective). Hemgenix is the first gene therapy to treat haemophilia B. It is delivered as a single infusion. LINK

CHMP recommends expansion of EU label for emicizumab to include people with moderate hemophilia A
December 16, 2022 — Roche announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended expansion of the emicizumab European Union (EU) marketing authorisation. If approved, the therapy would also be indicated for the routine prophylaxis of bleeding episodes in people with hemophilia A without factor VIII inhibitors who have moderate disease (FVIII ≥1% and ≤ 5%) with a severe bleeding phenotype. LINK.

U.S. Food and Drug Administration approves CSL’s HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B
November 22, 2022 — CSL today announced that the U.S. Food and Drug Administration (FDA) approved HEMGENIX® (etranacogene dezaparvovec-drlb), the first one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. LINK

Freeline takes difficult decision to not progress their hemophilia B program into Phase 3 development
November 15, 2022 — As a result of a previously announced evaluation of its investigational gene therapy program for hemophilia B, Freeline has decided to focus its resources on other programs, and stop investment in further development of it’s hemophilia B program without a partner. LINK

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC) SAN RAFAEL, Calif.
Aug. 24, 2022 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). LINK

Monkeypox Virus and Plasma Protein Therapies
27 June 2022 — PPTA considers that the current MPXV outbreak is not a concern for the safety margins of plasma protein therapies manufactured by PPTA member companies. This assessment is also shared by other concerned parties (AABB, WHO). PDF

Precision BioLogic Launches New Product to Improve Testing for Hemophilia B
MONTREAL – May 9, 2022 /CNW/ — Precision BioLogic Inc., a leading developer of hemostasis diagnostic products, is pleased to announce the launch of its new CRYOcheck Chromogenic Factor IX assay in Canada, the European Union, Australia, and New Zealand following market authorization by Health Canada and respective in-country regulatory authorities. The announcement was made at the World Federation of Hemophilia Congress taking place in Montreal, May 8–11, 2022. LINK

European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B
MARBURG, GERMANY – March 28, 2022 — Global biotherapeutics leader CSL Behring today announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for etranacogene dezaparvovec (EtranaDez) under its accelerated assessment procedure. LINK

BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A
SAN RAFAEL, Calif. – Mar. 17, 2022 /PRNewswire/, Ozelo, M. et al., Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A. N Engl J Med 2022; 386:1013-1025, DOI: 10.1056/NEJMoa2113708 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced publication of results from the Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in the New England Journal of Medicine (NEJM). LINK

Biomarin gives an update and reports a serious adverse event for its Phase 3 hemophilia A gene therapy study
Feb. 4, 2022 — An update for the hemophilia community from BioMarin, regarding the ongoing Phase 3 BioMarin hemophilia A gene therapy study, and a serious adverse event deemed, by the Independent Data Safety Monitoring Committee, unrelated to the investigational gene therapy from the phase 1/2 study. PDF

BioMarin Announces Stable and Durable Annualized Bleed Control in the Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A
SAN RAFAEL, Calif. – Jan. 9, 2022 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in hemophilia, with 134 participants. LINK

Sigilon Therapeutics Announces Strategic Reprioritization
CAMBRIDGE, Mass. – Dec. 13, 2021 (GLOBE NEWSWIRE) — Sigilon Therapeutics, Inc. (NASDAQ: SGTX), a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics platform, today announced a strategic reprioritization to enable the Company to focus on MPS-1 and diabetes. PDF

Sigilon Therapeutics Announces Update on SIG-001 Phase 1/2 Study in Hemophilia A
CAMBRIDGE, Mass. – Nov. 29, 2021 (GLOBE NEWSWIRE) — Sigilon Therapeutics, Inc. (NASDAQ:SGTX), a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics platform, today reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase 1/2 study of SIG-001 in severe or moderately severe hemophilia A. PDF

FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
November 5, 2021 — On Tuesday, November 4, the U.S. Food and Drug Administration (FDA) placed the Pfizer/Sangamo haemophilia A gene therapy programme, including the pivotal phase 3 AFFINE study ( NCT04370054 ), with giroctocogene fitelparvovec (SB-525 or PF-07055480), on clinical hold until the review of a proposed protocol amendment. ( Sangamo news release ). LINK

Spark Therapeutics’ SPK-8011 Suggests Stable and Durable Factor VIII Expression in Largest Phase 1/2 Gene Therapy Study in Hemophilia A to Date
PHILADELPHIA – July 21, 2021 — Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced updated data from the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A during the International Society of Thrombosis and Hemostasis (ISTH) 2021 Virtual Congress (July 17-21). LINK

European Medicines Agency Validates BioMarin's Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A
SAN RAFAEL, Calif. – July 15, 2021 (PRNewswire) — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. With today's validation the MAA review can now commence. A CHMP opinion is anticipated in the first half of 2022. LINK

uniQure Announces Positive 52-Week Clinical Data from HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B and Provides Regulatory Update
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – June 22, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. These are the first clinical data to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a single gene therapy investigational product to date. LINK

European Medicines Agency Grants BioMarin's Request for Accelerated Assessment of Valoctocogene Roxaparvovec for Treatment of Severe Hemophilia A
SAN RAFAEL, Calif. – May 24, 2021 (PRNewswire) — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Accelerated assessment reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP). A CHMP opinion is expected in the first half of 2022. LINK

CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure
KING OF PRUSSIA, PA, USA – May 6, 2021 — Global biotherapeutics leader CSL Behring today announced the closing of its global Commercialization and License agreement with uniQure (NASDAQ: QURE) for etranacogene dezaparvovec (AMT-061), a novel gene therapy for the treatment of hemophilia B. CSL Behring has been recognized as a global hematology leader for decades. The acquisition complements both CSL Behring’s cell and gene therapy scientific platform and its hematology product portfolio, which include other treatments for hemophilia B as well as therapies for treating hemophilia A, von Willebrand disease, thrombosis, and other life-threatening conditions. LINK

uniQure announces closing of commercialization and license agreement with CSL Behring
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – May 06, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired on May 5, 2021, and the agreement became fully effective today, May 6, 2021. The agreement was contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom. The antitrust review process is now complete in all three countries. PDF

uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – April 26, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced today that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company’s hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identified by the FDA related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial. PDF

uniQure Announces Findings from Reported Case of Hepatocellular Carcinoma (HCC) in Hemophilia B Gene Therapy Program
LEXINGTON, Mass. and AMSTERDAM, The Netherlands – March 29, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced today the results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec. The results of the investigation found that it is highly unlikely the HCC was caused by etranacogene dezaparvovec. LINK

Takeda Announces Strategic Partnership to Accelerate the Development of Enzyre’s Diagnostic Technology Platform That Aims to Improve the Standard of Care for Patients with Bleeding Disorders
OSAKA, JAPAN – March 4, 2021 — Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced it has entered into a strategic partnership with Enzyre to accelerate the development of Enzyre’s proprietary platform Enzypad to enable patients to test their blood coagulation in a home setting. The partnership builds on an existing research collaboration agreement with Enzyre signed in December 2019 and close collaboration over the last five years. LINK

Data at EAHAD 2021 Highlight Value of Takeda’s Leading Hematology Portfolio in Clinical Settings and Commitment to Patient-Focused Advancements
OSAKA, JAPAN – February 3, 2021 — Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced that 14 abstracts from the company’s Hematology portfolio and pipeline are being presented as electronic posters at the Virtual European Association for Haemophilia and Allied Disorders Congress (EAHAD 2021). Data include multiple abstracts reinforcing the long-term efficacy and safety of Takeda’s hematology portfolio in clinical practice, supporting the value of these treatments in a real world setting. PDF

BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set
SAN RAFAEL, U.S.A. – January 10, 2021 — BioMarin Pharmaceutical Inc. today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. LINK

UniQure Reports Development of Hepatocellular Carcinoma in a Gene Therapy Clinical Trial Participant
Lexington, MA and Amsterdam, the Netherlands – December 21, 2020 (BUSINESS WIRE) — UniQure, which is running a Phase 3 AAV5-Factor IX gene therapy study, reported today a case of hepatocellular carcinoma (HCC) in a trial participant1. HCC is a tumor that arises in the liver. LINK

Pfizer Doses First Participant in Phase 3 Study Evaluating anti-TFPI Investigational Therapy, Marstacimab, for People With Severe Hemophilia A and B With or Without Inhibitors
NEW YORK, U.S.A. – November 23, 2020 (BUSINESS WIRE) — Pfizer Inc. (NYSE: PFE) today announced that the first participant has been dosed in the Phase 3 BASIS study of marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor (anti-TFPI) being evaluated for the treatment of people with severe hemophilia A or B, with or without inhibitors. LINK

uniQure Announces Positive Top-Line Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B
LEXINGTON, Ma. – November 19, 2020 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive top-line data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. This is the first data set to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of patients receiving a single gene therapy investigational product to be reported to date. These clinical data were published today as a late-breaking abstract, one of only six accepted for presentation at the 62nd Annual Meeting of the American Society of Hematology (ASH) and will be featured as an oral presentation in the conference on December 8, 2020. LINK.

Takeda to Present Rare Bleeding Disorders Research at Thrombosis & Hemostasis Summit of North America (THSNA) 2020
CAMBRIDGE, Ma. and OSAKA, JAPAN – October 27, 2020 — Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”), today presented four abstracts at the Thrombosis and Hemostasis Summit of North America (THSNA), highlighting Takeda’s commitment to advancing personalized care in people living with bleeding disorders. LINK

rHEALTH’s FVIII Point-of-Care Device Awarded $1.5M Direct-to- Phase II Grant from the NIH
BEDFORD, Ma. – October 8, 2020 — rHEALTH LLC, a Diagnose Yourself, Anywhere™ company, today announced that its technology is the subject of the award from the National Institute of Health (NIH). The award is to rHEALTH’s affiliate, the DNA Medicine Institute (DMI), which has granted rHEALTH commercial rights to the technology. The focus of the grant is to further develop the company’s fingerstick-based point-of-care technology for monitoring both FVIII and emicizumab. PDF

An update from Roche on the evolution of Hemlibra post-approval data communications
September 23, 2020 — At Roche we recognise and appreciate the interest that the haemophilia community has in regards to Hemlibra (emicizumab), specifically around the subject of its long-term efficacy and safety, both now and in the future. We understand that the subject of long-term safety for haemophilia treatments is an important topic also for the community, and at Roche we will continue to listen to the needs of the community and aspire to provide information that leads to better clinical decision making and patient care. PDF

New England Journal of Medicine publishes positive final results from phase 1/2a study of BIVV001 in people with severe haemophilia A
Stockholm & Paris – September 10, 2020 — The New England Journal of Medicine today published positive final results from the phase 1/2a trial evaluating the safety, tolerability and pharmacokinetics of BIVV001 (rFVIIIFc-VWF-XTEN) in adult patients with severe haemophilia A. BIVV001 is an investigational factor VIII therapy designed to provide higher bleed protection in a once-weekly prophylactic treatment regimen. Sobi (STO:SOBI) and Sanofi collaborate on the development and commercialization of BIVV001. LINK

BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
SAN RAFAEL, Calif. – August 19, 2020 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A on August 18, 2020. The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. LINK

Precision BioLogic Inc. announces CRYOcheck™ Chromogenic Factor VIII Assay Cleared for Sale in U.S
HALIFAX – July 20, 2020 — Precision BioLogic Inc., a leading developer of hemostasis diagnostic products, is pleased to announce FDA 510(k) clearance and the launch of its new CRYOcheck Chromogenic Factor VIII assay in the U.S. This clearance follows authorizations received from regulatory authorities in Canada, the European Union, Australia, and New Zealand where the assay launched earlier this year. PDF

Takeda Provides Updates on Its Pharmacokinetic-guided Prophylaxis Studies at ISTH 2020, Reinforcing Its Commitment to Advancing Personalized Care for People with Bleeding Disorders
OSAKA, JAPAN – July 12, 2020 — Takeda Pharmaceutical Company Limited (“Takeda”) (TSE: 4502/NYSE:TAK) today announced that 13 abstracts are being presented from the company’s Hematology portfolio and pipeline at the International Society on Thrombosis and Haemostasis 2020 Virtual Congress (ISTH 2020). Among the data, Takeda spotlighted four abstracts to highlight its commitment to advancing personalized care through pharmacokinetic (PK)-guided prophylaxis in people living with hemophilia or von Willebrand Disease (VWD) – including scientific updates in patients with hemophilia A from the Phase 3 PROPEL and Phase 3b CONTINUATION studies investigating potential benefits of personalized TAK-660 (rurioctocog alfa pegol) prophylaxis. Two population studies into the PK/pharmacodynamic (PD) profiles of recombinant von Willebrand factor (rVWF), which provide data to assist in the optimization of rVWF personalized dosing strategies, were also presented. LINK

Spark Therapeutics Announces Updated Data on SPK-8011 from Phase 1/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress
PHILADELPHIA, Pa. – July 12, 2020 — Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced updated data from three dose cohorts of the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A. These data were presented at the International Society of Thrombosis and Hemostasis (ISTH) 2020 Virtual Congress by Principal Investigator Lindsey A. George, M.D., The Perelman School of Medicine, University of Pennsylvania and Children’s Hospital of Philadelphia. LINK

CSL Behring Agrees to Acquire Novel Late-Stage Gene Therapy Candidate for Hemophilia B Patients from uniQure
KING OF PRUSSIA, Pa. – June 24, 2020 — Global biotherapeutics leader CSL Behring announced today that it has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure (NASDAQ: QURE), a leading gene therapy company. The AMT-061 program, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B. LINK

BioMarin Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
SAN RAFAEL, Calif. – May 31, 2020 — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. The data have been submitted as a late-breaking abstract to the World Federation of Hemophilia (WFH) Virtual Summit to be held June 14-19, 2020. LINK

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Disclaimer

The information on the WFH website is provided for general information purposes only. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.

While every effort has been made to ensure the accuracy of the information on this site, the WFH does not guarantee the information is accurate, and is not responsible in any way whatsoever for damages arising out of the use of this website or any of the information contained herein.

Messages posted to WFH discussion forums, Facebook, Twitter, and other social media platforms do not represent the opinions of the World Federation of Hemophilia, its staff, or Board of Directors. The author of a message is solely responsibility for its content. Information posted on WFH social networks and platforms should never be a substitute for individualized professional medical advice, even when the author has medical qualifications or is considered an authority. Information posted to a discussion group should not be used to diagnose or treat a specific health problem without consulting a qualified healthcare professional. The WFH recommends that you contact your physician or local treatment centre if you have any individual questions or concerns.

References and links to other websites or references to other organizations, products, services, or publications do not constitute endorsement or approval by the WFH. The WFH is not responsible and assumes no liability for the content of any linked websites.

Fraud Alert

Unauthorized solicitations – Warning

The WFH has been made aware of various correspondences—circulated via e-mail and telephone—coming from individuals impersonating WFH staff or falsely stating that they are associated with the WFH. These correspondences, which may seek to obtain money using the name of someone affiliated with the WFH, are fraudulent and come from outside of our organization.

If you receive a suspicious solicitation, exercise extreme caution. In the case of an email, look at the email address to see if it looks suspicious (for example, all WFH emails come from @wfh.org).

We are asking you to remain vigilant, and if you have any doubts about the correspondence, please forward the email to the WFH at [email protected] or call +1 514-875-7944.

Hemophilia Alliance

Recognizing that training women leaders ensures the diversity our community need to thrive; the Hemophilia Alliance has been a longtime supporter of the Susan Skinner Memorial Fund. They deepened their engagement with the global community through the establishment of the Travel Grant Program, which allows US-based multidisciplinary healthcare professionals the opportunity to attend WFH global training workshops. The wealth of experience and perspective attendees bring back home to their communities helps patients across the country. Their patience and support as we grappled with changing norms in these challenging times gave us the flexibility to meet our community’s needs with revamped programming.

India - Mumbai

Year of IHTC designation: 2000

National Institute of Immunohaematology (ICMR) and KEM Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Dental care
  • Nursing
  • Orthopedic care
  • Physiotherapy

India - Vellore

Year of IHTC designation: 2000

Christian Medical College, Vellore

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics with antenatal diagnosis
  • Nursing
  • Musculoskeletal management – physical and occupational therapy with physiatry
  • Orthopedic care and surgery
  • Blood banking
  • Dental care
  • Psychology/Social services
  • Antenatal diagnosis

Senegal - Dakar

Year of IHTC designation: 2016

Centre International de Traitement de l’Hémophilie (CITH) de Dakar
Centre National de Transfusion Sanguin (CNTS)

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Blood banking
  • Physiotherapy

Russia - Moscow and St. Petersburg

Year of IHTC designation: 2010

National Medical Research Center of Hematology of the Russian Academy of Medical Sciences

Areas of Specialty

  • Adult hematology
  • Nursing
  • Orthopedic surgery

Netherlands - Utrecht

Year of IHTC designation: 1996

Van Creveldkliniek
University Medical Centre Utrecht

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hematology
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy/Occupational therapy
  • Psychology and social services

Japan - Nara

Year of IHTC designation: 1998

Nara Hemophilia Centre
Nara Medical University

Areas of Specialty

  • Adult and pediatric hematology
  • Dental care
  • Laboratory diagnosis and Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy

Italy - Milan

Year of IHTC designation: 1972

Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking
  • Dental care
  • Diagnostic assessment of platelet functional defects
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology and social services

Israel - Tel Hashomer

Year of IHTC designation: 1993

National Hemophilia Center, Tel Hashomer
Sheba Medical Center

Areas of Specialty

  • Adult and pediatric hematology
  • Orthopedic surgery
  • Nursing
  • Physiotherapy
  • Dental care
  • Laboratory diagnosis (not genetics)
  • Psychology

France - Paris

Year of IHTC designation: 1972

Paris Haemophilia Centre (Necker (N) and Kremlin Bicêtre (KB) Hospitals)

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Dental care
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology and Social services

South Africa - Johannesburg

Year of IHTC designation: 2016

Haemophilia Comprehensive Care Centre
Charlotte Maxeke Johannesburg Academic Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Dental care
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic surgery
  • Physiotherapy

Egypt - Cairo

Year of IHTC designation: 2015

Shabrawishi Hospital, Egyptian Society of Hemophilia, and Cairo University

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Molecular genetics
  • Blood banking
  • Physiotherapy

Canada - Toronto

Year of IHTC designation: 2018

University of Toronto Hemophilia Centres
St. Michael’s Hospital

Areas of Specialty

  • Adult hematology services
  • Pediatric hematology services
  • Physiotherapy/occupational therapy
  • Dental care
  • Orthopaedic care (no surgery)
  • Laboratory diagnosis
  • Nursing
  • Psychology/social services

Brazil - Campinas, SP

Year of IHTC designation: 2008

Hemocentro – “Unidade de Hemofilia Cláudio Luiz Pizzigatti Corrêa”
Hemocentro Unicamp, University of Campinas

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and molecular genetics
  • Nursing
  • Dentistry
  • Orthopedic care and surgery
  • Physiotherapy

Belgium - Brussels

Year of IHTC designation: 2013

Haemostasis and Thrombosis Unit
Division of Haematology
Cliniques universitaires Saint-Luc

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis
  • Orthopedic care and surgery
  • Physiotherapy

Austria - Vienna

Year of IHTC designation: 1972

Medical University of Vienna
Department of Medicine I
Clinical Division of Hematology and Hemostaseology

Areas of Specialty

  • Adult and pediatric hematology

Australia - Melbourne

Year of IHTC designation: 2005

Ronald Sawers Haemophilia Treatment Centre

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis and hemostasis
  • Orthopedic care and surgery
  • Nursing
  • Dental care
  • Blood banking
  • Psychology
  • Social work

Argentina - Buenos Aires

Year of IHTC designation: 1974

Instituto Asistencial “Dr. Alfredo Pavlovsky”
(Fundación de la Hemofilia)

Instituto De Investigaciones Hematológicas “Dr. Mariano R. Castex” (IIHEMA
(Academia Nacional de Medicina)

Areas of Specialty

  • Adult and pediatric hematology
  • Physiotherapy
  • Dental care
  • Orthopaedic care and surgery
  • Laboratory diagnosis and genetic diagnosis
  • Nursing
  • Psychology
  • Social services

Singapore - Singapore

Year of IHTC designation: 2002

Singapore General Hospital Haemophilia Treatment Clinic

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis
  • Nursing
  • Dental care
  • Orthopedic care (no surgeries)
  • Physiotherapy
  • Psychology

Spain - Madrid

Year of IHTC designation: 2018

Hospital Universitario La Paz

Areas of Specialty

  • Adult and pediatric hematology
  • Physiotherapy/occupational therapy
  • Dental care
  • Orthopaedic care (no surgery)
  • Laboratory diagnosis
  • Nursing
  • Psychology/social services

Spain - Valencia

Year of IHTC designation: 1997

Haemostasis and Thrombosis Unit
Haematology Service, University and Polytechnic Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Blood banking
  • Psychology and Social services

Hemophilia of Georgia

Hemophilia of Georgia has stood with us for over three decades, playing an early role in distributing donated factor via the WFH Humanitarian Aid program. Our relationship has deepened over the years as they continued to support this program and came on board as a founding Chapter Challenge champion in 2016. Their belief in educating future leaders is clear in their investment in the Youth Leadership Workshops, which has led to the training of over 50 young adults. They also support workshops devoted to educating providers and patient leaders on how to collect and use data to advocate for better patient care. Their staff generously volunteer their time as trainers and as well as serving on various committees.

Twins of the Year Awards

2022 Amman (Jordan) – Dublin (Ireland)
2021 Addis Ababa (Ethiopia) – Minneapolis (U.S.A) and
Tehran (Iran) – Milan (Italy)
2020 Aluva (India) – Newcastle (UK)
2019 Yogyakarta / Solo (Indonesia) – Utrecht (Netherlands)
2018 Abidjan (Ivory Coast) – Brussels (Belgium)
2017 Santa Cruz de la Sierra (Bolivia) – Buenos Aires (Argentina)
2016 Kampala (Uganda) – London (UK)
2015 Varna (Bulgaria) – Bonn (Germany)
2014 Arequipa (Peru) – Los Angeles (USA)
2013 Davangere (India) – Liverpool (UK)
Ludhiana (India) – Detroit (USA)
2012 Hanoi (Vietnam) – Melbourne (Australia)
2011 Manado (Indonesia) – Utrecht (Netherlands)
2010 Delhi (India) – Winnipeg (Canada)
Yaoundé (Cameroon) – Geneva (Switzerland)
2009 Chisinau (Moldova) – Warsaw (Poland)
Colombo (Sri Lanka) – Vellore (India)
2008 Gaborone (Botswana) – Philadelphia (USA)
2007 Vientiane (Laos) – Brest (France)
Damascus (Syria) – Montpellier (France)
2006 Lima (Peru) – Fort Worth (USA)
2005 Yerevan (Armenia) – Minneapolis (USA)
Casablanca (Morocco) – Caen (France)
2004 Beirut (Lebanon) – Geneva (Switzerland)
Santo Domingo (Dominican Republic) – Caracas (Venezuela)
2003 Cairo (Egypt) – Knoxville (USA)
2002 Moscow (Russia) – Liverpool (UK)
Panama – Valencia (Spain)
2001 Bangalore (India) – St. Louis (USA)
2000 Pune (India) – Bradford (UK)
1999 Montevideo (Uruguay) – Buenos Aires (Argentina)
Tianjin (China) – Calgary (Canada)
1998 Plovdiv (Bulgaria) – Bonn (Germany)
1997 Bogota (Colombia) – Los Angeles (USA)
Tartu (Estonia) – Stockholm (Sweden)
1996 Timisoara (Romania) – Munich (Germany)
Riga (Latvia) – Munster (Germany)
1995 Klaipeda (Lithuania) – Malmo (Sweden)
1994 Bratislava (Slovakia) – Tel Hashomer (Israel)

National Bleeding Disorders Foundation

The National Bleeding Disorders Foundation helps advance our mission on several fronts. They inspire others to follow their lead as a founding champion of the NHF Chapter Challenge. Their commitment to equality of access is demonstrated in their support of the Cornerstone Initiative, which lays the foundation for care in the world's most underserved regions, as well as programs that aim to raise awareness and improve care of women with bleeding disorders and those with von Willebrand disease. We also benefit from their steadfast engagement with various programs including the WFH Twinning Program and their individual leaders’ contribution to our leadership and committees.

Legacy Gift

Do you sometimes wish you could do more? A bequest is a simple way for you to make a significant gift without changing anything about your financial security and lifestyle today. Your legacy will sustain care for the generations to come.

Download these sample clauses to give to your legal advisor.

We understand if you prefer to remain anonymous, but we would be very honoured if you would let us know of your intentions so that we may thank you personally. We would also be happy to answer any questions you may have or give you additional information. Please contact Jennifer Laliberté at 514‑875‑7944 ext.2822 or [email protected].

Disclaimer

The information on the WFH USA website is provided for general information purposes only. The WFH USA does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH USA recommends that you contact your physician or local treatment centre. Before administering any products, the WFH USA urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.

While every effort has been made to ensure the accuracy of the information on this site, the WFH USA does not guarantee the information is accurate, and is not responsible in any way whatsoever for damages arising out of the use of this website or any of the information contained herein.

Messages posted to WFH USA discussion forums, Facebook, Twitter, and other social media platforms do not represent the opinions of the World Federation of Hemophilia, its staff, or Board of Directors. The author of a message is solely responsibility for its content. Information posted on WFH USA social networks and platforms should never be a substitute for individualized professional medical advice, even when the author has medical qualifications or is considered an authority. Information posted to a discussion group should not be used to diagnose or treat a specific health problem without consulting a qualified healthcare professional. The WFH USA recommends that you contact your physician or local treatment centre if you have any individual questions or concerns.

References and links to other websites or references to other organizations, products, services, or publications do not constitute endorsement or approval by the WFH USA. The WFH USA is not responsible and assumes no liability for the content of any linked websites.

Website privacy policy

The WFH USA does not collect personally identifying information about you when you visit our site, unless you choose to provide such information to us. Providing such information is strictly voluntary. This policy is your guide to how we will handle information we learn about you from your visit to our website.

Use of links

Throughout our website, we provide links to other servers which may contain information of interest to our readers. We take no responsibility for, and exercise no control over, the organizations, views, or accuracy of the information contained on other servers. When linking to the WFH USA, we request that you ensure that there are no associated connections for commercial purposes. Any official use of the name WFH USA or the use of its logo needs to be approved by the WFH USA. If you have a link you’d like us to consider adding to our website, please send an email to [email protected] with the subject “Link request.”

Use of text and images

All information, photographs, graphics, and other material on this website are protected by copyright. While users of this website may use the content for personal, non-commercial use, none of the content of this website can be modified, translated, transferred or sold, copied, reproduced, published, or distributed, in whole or in part, without the prior written consent of the WFH USA. To view a copy of the WFH USA’s permissions guidelines, click here.

The WFH USA encourages the dissemination of its information and welcomes proposals on the reprinting, redistribution, and translation of its materials. If you would like to publish information that you find on our website, please send your request to [email protected].

Accessibility

If you have suggestions on how to make the site more accessible, please contact us at [email protected].

Opt-Out or Change Your Contact Information

Our website provides users the opportunity to opt-out of receiving communications from us through a special online form or via the communication they have received. You may, at all times, choose to receive which communications within the organization you can receive or none at all. You may also update your contact information previously provided to us via another online form.

Questions about our policies

If you have any questions about this privacy statement, the practices of this site, or your dealings with this website, you can contact us at [email protected].

Fraud alert

Unauthorized solicitations – Warning

The WFH USA has been made aware of various correspondences—circulated via e-mail and telephone—coming from individuals impersonating WFH USA staff or falsely stating that they are associated with the WFH USA. These correspondences, which may seek to obtain money using the name of someone affiliated with the WFH USA, are fraudulent and come from outside of our organization.

If you receive a suspicious solicitation, exercise extreme caution. In the case of an email, look at the email address to see if it looks suspicious (for example, all WFH USA emails come from @wfhusa.org or @wfh.org).

We are asking you to remain vigilant, and if you have any doubts about the correspondence, please forward the email to the WFH USA at [email protected] or call 1-877-417-7944.

International Award for Outstanding Service - Recipients

outstanding-service-award-2020
outstanding-service-award-2010

International Healthcare Volunteer Award - Recipients

healthcare-award-2022-en
healthcare-award-2020
healthcare-award-2018
healthcare-award-2016
healthcare-award-2014

Previous years

2012 Piet de Kleijn
The Netherlands
2010 Dr. Mammen Chandy
India
2008 Dr. Man-Chiu Poon
Canada
2006 Dr. Norma de Bosch
Venezuela
2004 Dr. Parttraporn Insarangkura
Thailand

Dr. Carol K. Kasper
United States

Pietrogrande Prize

2023 Nicholas Goddard
United Kingdom
2021 Axel Seuser
Germany
2019 Dr. Adolfo Llinás
Colombia
2017 Pamela Hilliard
Canada
2015 Kathy Mulder
Canada
2013 James Luck
United States
2012 Piet de Kleijn
The Netherlands
2011 Dr. Lily Heijnen
The Netherlands
2010 Dr. Horacio Caviglia
Argentina
2009 Jerome D. Wiedel
United States
2008 Dr. Federico Fernández-Palazzi
Venezuela
2007 Michael Heim
Israel
2006 Brenda Buzzard
United Kingdom
2005 Dr. Marvin Gilbert
United States

Sweden - Malmö

Year of IHTC designation: 1976

Malmö Centre for Thrombosis and Haemostasis
Department for Hematology and Coagulation Disorders, Skåne University Hospital
Skåne University Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking (on request)
  • Dental care and surgery
  • Laboratory diagnosis and genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy

Henri Horoszowski Award

2021 Baolai Hua
Yangzhou College, Xuzhou Medical University		 The effects of intra-articular injection of TNF-alpha Antagonists in treatment of haemophilic synovitis
2019 Maarten Eerdekens
Belgium		 Association between multi-segment foot kinetics and blood induced joint damage in adult patients with hemophilic ankle arthropathy
2017 Fabio Souza
Brazil		 Decrease in joint inflammation, swelling and pain after a swimming protocol in animal model of experimental-induced hemarthrosis
2015 SM Javd Mortazavi
Iran		 Bilateral total knee arthroplasty in patients with hemophilia: A safe and cost-effective procedure
2013 Lize F.D. van Vulpen
United States		 A fusion protein of IL4 and IL10, is equally effective in protecting cartilage from blood-induced damage compared to the individual components
2012 Horacio Caviglia
Argentina		 Pseudotumor Surgery in Haemophilia A patients: Comparitive results between inhibitor and non-inhibitor patients
2011 Nick Goddard
United Kingdom and
Monique van Meegeren
The Netherlands		 Early onset administration of IL-4 and IL-10 prevents blood-induced cartilage damage in vitro
2010 Lydia Abad-Franch
Spain		 Influence of an Aquatic Training Protocol on the Reduced Risk of Cardiovascular Morbidity and Mortality in a Hemophiliac
2009 Sebastien Lobet
Belgium		 Impact of Hemophilia Ankle Arthropathy on Gait Disability: Analysis of Energetic and Mechanical Variables
2008 Jose Alberto Tlacuilo-Parra
Mexico		 Bone Turnover Markers and Bone Mineral Density in Children with Hemophilia
2007 Natalie Jansen
The Netherlands		 Protective Abilities of Interleukin-10 in Blood-induced Cartilage Damage
2006 Axel Seuser and E. Kusch
Germany		 Multi-Center Study of Orthopedic Outcome of the Lower Extremities in 249 Children with Hemophilia
2005 Frank van Genderen, Nico van Meeteren, Lily Heijnen, Piet de Kleijn, Marijke van den Berg, Paul Helders
The Netherlands		 The Hemophilia Activities List: Final development and validation of a hemophilic-specific self-assessment questionnaire on functional abilities
2004 Goris Roosendaal, Zalima N Jahangier, Kim MG Jacobs, Johannes WJ Bijlsma, Floris PJG Lafeber
The Netherlands		 Radiosynoviorthesis using YTTRIUM-90 results in adverse direct effects on cartilage: a human in vitro study
2003 Axel Seuser, Thomas Wallny, Günter Schumpe, Elmar Trunz, Hans-Hermann Brackman
Germany		 How to advise young haemophiliacs to find the right sport? A new and safe algorithm
2002 James Luck, Jr. and Mauricio Silva
United States		 Long-Term Review of Total Knee Arthroplasty
2001 Federico Fernández-Palazzi
Venezuela		 Oxytetracycline Chlorohydrate: A new material for chemical synovial orthoses
2000 Horacio Caviglia
Argentina		 Pseudotumours
1999 William J. Ribbans and J.L. Hicks
United Kingdom		 Communication and Co-operation for the New Millennium – The Future of the Musculoskeletal Committee in the 21st Century
1998 Adolfo Llinas, Mauricio Silva, Monica Duarte, Gonzalo Ucros, Graciela Perini, Angela Maria Cerquera, Andres Forero
Colombia		 Chromic phosphate synoviorthesis without age restriction should be the first treatment option for chronic active hemophilic synovitis

International Frank Schnabel Volunteer Award - Recipients

ifs-award-2022-en
ifs-award-2020
ifs-award-2018
ifs-award-2016
ifs-award-2014

Previous years

2012Martha Patricia Monteros Rincon
Mexico
2010Antonia Luque de Garrido
Venezuela
2008Jean Michel Bouchez
France
2006Yuri Zhulyov
Russia
2004Ashok Verma
India

Functional Scales

The outcomes of interest collected by the WBDR include demographic characteristics, clinical and treatment-related outcomes, burden of disease outcomes, and patient-reported outcomes.

The WBDR includes five functional scales as part of its extended data set:

WBDR hemophilia data sets

WBDR Minimal Data Set

The WBDR Minimal Data Set (MDS) is a set of data on demographics, diagnosis, symptoms and treatment which are collected on patients participating in the WBDR.

WBDR Extended Data Set

The WBDR Extended Data Set (EDS) is a larger set of data, with more details than the MDS, which includes questions on patient demographics, diagnosis, symptoms, treatment and burden of disease. The EDS can be used by hemophilia treatment centers to collect data on patients participating in the WBDR. The WBDR Extended Data Set (EDS), was added to the database in March 2019.

United States - Columbus, Ohio

Year of IHTC designation: 2018

Nationwide Children’s Hospital

Areas of Specialty

  • Dental care
  • Laboratory diagnosis
  • Nursing
  • Orthopedic care and surgery
  • Pediatric hematology services
  • Physiotherapy/occupational therapy
  • Psychology/social services

United States - Houston, Texas

Year of IHTC designation: 2000

Gulf States Hemophilia and Thrombophilia Center
University of Texas Health Science Center at Houston

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Blood banking
  • Physiotherapy/ Occupational therapy
  • Psychology/Social services

United States - Los Angeles, California

Year of IHTC designation: 1970

Lifespan Orthopaedic Hemophilia Treatment Center at Luskin Orthopedic Institute for Children

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology/Social services

United States - New York, New York

Year of IHTC designation: 1993

Mount Sinai School of Medicine
Mount Sinai Medical Center

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Molecular genetics
  • Social services

United States - Rochester, Minnesota

Year of IHTC designation: 1974

Mayo (Rochester) Comprehensive Hemophilia Center, and International Hemophilia Training Center (WFH)
Mayo Clinic/Foundation and Mayo Medical School

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology/ Social services

United States - San Diego, California

Year of IHTC designation: 2018

Hemophilia & Thrombosis Treatment Center
University of California San Diego

Areas of Specialty

  • Adult hematology
  • Physiotherapy/occupational therapy
  • Dental care
  • Orthopaedic care (no surgery)
  • Laboratory diagnosis
  • Nursing
  • Psychology/social services

United Kingdom - London

Year of IHTC designation: 1970

Katharine Dormandy Haemophilia Centre and Thrombosis Unit
Royal Free Hospital

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy/Occupational therapy
  • Molecular genetics
  • Psychology/Social services

United Kingdom - Oxford

Year of IHTC designation: 1972

Oxford Haemophilia and Thrombosis Centre
Churchill Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Orthopedic care and surgery
  • Nursing
  • Molecular genetics
  • Physiotherapy/Occupational therapy
  • Psychology and social services

United Kingdom - Sheffield

Year of IHTC designation: 1992

Sheffield Haemophilia and Thrombosis Centre
Royal Hallamshire Hospital

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics

Thailand - Bangkok

Year of IHTC designation: 1984

International Hemophilia Training Center – Bangkok
Ramathibodi Hospital
Mahidol University

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking
  • Dental care and surgery
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology and social services

Our history

The WFH was established in 1963 by Frank Schnabel, a Canadian businessman born with severe hemophilia A. He believed that patient organizations could be much more effective—and do more to represent the interests of people living with bleeding disorders—if they worked together. The first WFH Congress was held in Copenhagen, Denmark, on June 25, 1963, and was attended by representatives from 12 countries. Now, the WFH and our global network of national member organizations (NMOs) represent the interests of people with hemophilia and other inherited bleeding disorders in 152 nations.