GENE THERAPY
Overview
Hemgenix is a prescription medication approved by the U.S. Food and Drug Administration (FDA) to treat adults with hemophilia B. To be eligible for Hemgenix, you must either currently be using factor IX (9) prophylaxis therapy, have a life-threatening bleed or a history of one, or have repeated, serious, spontaneous bleeding episodes. Hemgenix is also known by its drug name, etranacogene dezaparvovec-drlb.
Hemgenix, a gene-replacement therapy, is believed to work by providing the gene for clotting factor IX, which is missing in people with hemophilia B. Hemgenix is delivered via an adeno-associated virus vector.
How do I take it?
Hemgenix is given as a one-time intravenous infusion.
Side effects
The FDA-approved label for Hemgenix lists common side effects including headache, flu-like symptoms, infusion-related reactions, fatigue, malaise (feeling unwell), and changes in certain blood test results.
Rare but serious side effects listed for Hemgenix include severe infusion reactions, liver damage, and the potential for developing liver cancer in people with preexisting risk factors.
For more details about this treatment, visit:
Hemgenix — CSL Behring
https://www.hemgenix.com
FDA Approves First Gene Therapy To Treat Adults With Hemophilia B — U.S. Food and Drug Administration
https://www.fda.gov/news-events/press-announcem...