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WFH statement - EN

Primer fármaco de terapia génica para la hemofilia B aprobado por la FDA

La Administración de Alimentos y Medicamentos de Estados Unidos (FDA por su sigla en inglés) aprobó el etranacogene dezaparvovec-drlb (HEMGENIX), la primera terapia génica de administración única para adultos con hemofilia B que reúnen los requisitos para recibirla. La terapia fue aprobada para el tratamiento de adultos con hemofilia B que actualmente utilizan terapia profiláctica con factor IX o que tienen un historial actual o previo de hemorragias que ponen en peligro la vida, o que han tenido episodios hemorrágicos espontáneos graves y repetidos.

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El etranacogene dezaparvovec-drlb permite a los adultos con hemofilia B elegibles que recibieron tratamiento con la terapia génica producir su propio factor IX, lo que puede reducir el riesgo de hemorragias y reducir o eliminar la necesidad de tratamiento profiláctico. En el ensayo clínico en curso, el tratamiento redujo la tasa de hemorragias anuales y el 94 por ciento de los pacientes (51 de 54) suspendieron la profilaxis con factor IX y permanecieron sin recibir profilaxis. Los efectos secundarios más comunes (incidencia ≥5%) fueron aumento de las enzimas hepáticas, dolor de cabeza, concentraciones elevadas de una enzima sanguínea determinada, síntomas similares a los de la influenza, reacciones relacionadas con la infusión, cansancio, náusea y malestar.

Para leer el comunicado de prensa de la FDA (en inglés) haga clic aquí.

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