First Gene Therapy for Hemophilia B is Approved by the FDA

The Coalition for Hemophilia B is pleased to join the rest of the community in celebrating the U.S. Food and Drug Administration (FDA)’s approval of the first gene therapy for hemophilia B according to the manufacturer, CSL Behring. The product with the scientific name of etranacogene dezaparvovec-drlb, will be marketed with the name of HEMGENIX®. etranacogene dezaparvovec-drlb. This historic new product is designed to reduce or eliminate bleeding in adults for years following a single infusion. In clinical trials, HEMGENIX reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free.

“We are thrilled to witness this milestone in hemophilia B treatment,” shared Kim Phelan, Chief Operating Officer of The Coalition for Hemophilia B. “Over the years we have seen a variety of advancements for the hemophilia community, but gene therapy is the first treatment option to offer those living with hemophilia B--and caregivers--the possibility of freedom from the need for regular, ongoing infusions.”

“As a man with hemophilia B and a community leader, I like others have been waiting for a treatment like this for much of our lives,” said Wayne Cook, President of the Coalition. “This is truly exciting news” Cook said.

“As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B,” said Paul Perreault, CSL’s Chief Executive Officer and Managing Director. “We recognize and thank all trial participants, scientists and investigators—without whom this important achievement would nothave been possible—and look forward to seeing the positive impact of HEMGENIX on the hemophilia B community.”

“HEMGENIX is unique in its approach to increasing mean factor IX activity and hemostatic protection in those with hemophilia B, and today’s approval could fundamentally transform the treatment paradigm for this life-long condition,” said Dr. Steven Pipe, Professor and the Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology at the University of Michigan and a lead investigator in the study that led to the drug, as quoted in CSL’s press release. “As a clinician, I look forward to being able to provide a new treatment option that may help patients treated with HEMGENIX become free from the regular infusion schedule that many people living with hemophilia B rely on to protect them from the debilitating effects of the condition.”

“It is an honor to be an advocate for this process and to educate our community about it” said Dr. David Clark, a scientist and Chairman of the Coalition. “This marks a new era in the treatment of hemophilia and many other conditions” he said.

You can read CSL Behring’s press release with more details about HEMGENIX at cslbehring.com