Global biotechnology leader CSL (ASX: CSL) has announced that the U.S. Food and Drug Administration (FDA) approved Hemgenix (etranacogene dezaparvovec-drlb), the first and only onetime gene therapy for appropriate adults with hemophilia B.

• Hemgenix is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.
• Hemgenix®, etranacogene dezaparvovec-drlb is administered as a single intravenous infusion and can be administered only once.
• In the ongoing clinical trial, Hemgenix reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free.
• If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.
• The most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell.
• Read more… https://www.drugs.com/newdrugs/fda-approves-hemgenix-etranacogene-dezaparvovec-drlb-genetherapy-hemophilia-b-5931.html Tzield November 17, 2022 – Provention Bio, Inc. (Nasdaq: PRVB),