Hemgenix® is First Gene Therapy Approved by FDA as Treatment for Hemophilia B

The Hemophilia community is celebrating strides in treatment availability following recent FDA approval of Hemgenix® (etranacogene dezaparvovec-drlb), the first gene therapy for treating hemophilia B.

Hemophilia B, primarily expressed in males, is a rare, inherited and lifelong bleeding disorder caused by a single gene defect that reduces the production of a protein called clotting factor IX (FIX). A single dose intravenous infusion of Hemgenix reduces the rate of abnormal bleeding by enabling the body to continuously produce FIX.

The Institute of Clinical and Economic Review (ICER) estimates that approximately 860 patients in the U.S. are eligible for this gene therapy treatment based upon the FDA-approved indication with 20% initiating treatment in each of the next 5 years.

Hemgenix will cost $3.5 million, making it the most expensive medication approved to date. This price does not consider treatment-related costs or those related to patient monitoring.

To learn more about hemophilia B and Hemgenix®, click here.