FDA approves Hemgenix, the first gene therapy to treat adults with hemophilia B

The US Food and Drug Administration (FDA) has approved Hemgenix (etranacogene dezaparvovec) gene therapy for the treatment of adults with hemophilia B (congenital factor 9 deficiency) who are currently using prophylactic factor 9 therapy; have current or historical life-threatening bleeding; or have repeated severe episodes of spontaneous bleeding.

Despite advances in the treatment of hemophilia, prevention and treatment of bleeding episodes can negatively impact people’s quality of life, said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in a press release. “Today’s approval provides a new treatment option for patients with haemophilia B and represents an important advance in the development of innovative therapies for those experiencing a high disease burden associated with this form of haemophilia,” he said.

The approval could dramatically transform the treatment paradigm for this lifelong condition, said Steven Pipe, MD, a professor at the University of Michigan and principal investigator in the HOPE-B study, in a company news release. “As a physician, I look forward to being able to provide a new treatment option that can help Hemgenix-treated patients break free from the normal infusion schedule that many people living with haemophilia B rely on to protect themselves from the effects debilitating condition,” he said.

While there are still things we’re learning about Hemgenix for hemophilia B, especially over the long term, the data we have so far has been very encouraging, says Nigel Key, MBChB, professor at the UNC School of Medicine and director of the UNC hemophilia and thrombosis center in Chapel Hill, NC.

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