Hemophilia gene therapy Hemgenix sets record for world’s most expensive drug 

FDA has approved CSL Behring’s (ASX: CSL) Hemgenix (etranacogene dezaparvovec), an adenovirus-associated virus–based gene therapy for adults with hemophilia B.

The price tag for the gene therapy — the first for hemophilia B — is $3.5 million. The drug is administered as a single infusion.

Hemophilia B is a genetic bleeding disorder caused by missing or defective blood coagulation factor IX, a clotting protein. The condition affects men more often than women.

People with moderate to severe hemophilia B typically receive regular prophylactic infusions of factor IX replacement therapy, frequently several times per week.

In the Phase 3 HOPE-B study, Hemgenix demonstrated “a profound and impressive decrease in bleeding rates compared to standard of care,” said Dr. Steve Pascoe, CSL Behring’s senior vice president of clinical and therapeutic strategy, in an R&D investor briefing.

“We also saw that factor IX level…

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