CSL Behring gets Hemgenix FDA approval for hemophilia B

CSL Behring, a US-based biotechnology company, has received approval for its Hemgenix (etranacogene dezaparvovec-drlb) from the US Food and Drug Administration (FDA) as a one-time gene therapy for hemophilia B in adults.

Hemophilia B is a bleeding disorder resulting from a single gene defect, which causes inadequate production of factor IX. Produced mainly by the liver, Factor IX is a protein that helps in blood clotting.

Hemgenix FDA approval was based on the findings of the ongoing HOPE-B clinical trial in which the gene therapy candidate reduced the rate of annual bleeds and eliminated the need for prophylactic therapy, while generating increased and sustained factor IX levels in 94% (51 out of 54) patients.

Paul Perreault — CSL Behring CEO and Managing Director, commenting on Hemgenix FDA approval, said: “… we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B.

“We recognize and thank all trial participants, scientists and investigators—without whom this important achievement would not have been possible—and look forward to seeing the positive impact of Hemgenix on the hemophilia B community.”

CSL Behring gets Hemgenix FDA approval for hemophilia B. Image courtesy of PublicDomainPictures from Pixabay.

uniQure led the multi-year clinical development program for Hemgenix while CSL Behring funded the clinical trials after it acquired global rights to commercialize the treatment.

Matt Kapusta — uniQure CEO said: “We have always believed that gene therapy had the potential to provide transformative benefits to people living with hemophilia B and are excited that the hemophilia community will have a new, safe and effective treatment option available to them.”

CSL Behring, a CSL business, said that it would make the Hemgenix gene therapy for hemophilia B available for eligible patients at the earliest.

Hemgenix is still currently under assessment by other regulatory agencies, according to the biotech company.

The FDA, in a statement, said Hemophilia B affects approximately one in 40,000 people and accounts for nearly 15% of patients with the disease.