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Landmark approval for first haemophilia B gene therapy

Words by GOLD newsdesk

In a landmark moment for people living with haemophilia B, the first-ever gene therapy for the condition has been approved by the FDA.

CSL Behring’s drug Hemgenix is a one-time therapy for the disease that works by shrinking the rate of annual bleeds, reducing or eliminating the need for prophylactic therapy, and generating elevated and sustained factor IX levels for years after infusion. In the clinical trial, 94% of patients discontinued factor IX prophylaxis and remained prophylaxis-free after being administered with Hemgenix.

“Gene therapy for haemophilia has been on the horizon for more than two decades,” said Peter Marks, Director, Center for Biologics Evaluation and Research, FDA. “This approval provides a new treatment option for patients with Haemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease.”

Currently, people with haemophilia B deal with challenging disease management pathways, largely controlling the condition with frequent blood infusions in the hospital setting, which can create burden for the patient themselves, but also for their family and caregivers.

“We are thrilled to witness this milestone in haemophilia B treatment,” commented Kim Phelan, Chief Operating Officer, The Coalition for Hemophilia B. “Gene therapy is the first treatment option to offer those living with haemophilia B – and caregivers – the possibility of freedom from the need for regular, ongoing infusions.”

Haemophilia B is the rarer of the two types of haemophilia and occurs when there is missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding. The disease affects one in 40,000 people in the US – primarily men – and makes up 15% of the total number of patients with haemophilia in the country.

Hear Laurence Woollard, Founder and Director, On the Pulse Consultancy, and haemophilia patient advocate, give his verdict on the FDA approval on this week's GOLD Podcast.

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