Drugs

New Hemophilia B Gene Therapy Approved

FDA has approved Behring’s Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy to treat adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current of historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. An agency notice says Hemgenix is a one-time gene therapy product given as a single dose by IV infusion. Its safety and effectiveness were evaluated in two studies of 57 adult men with severe or moderately severe Hemophilia B. Effectiveness was established based on decreases in the men’s annualized bleeding rate, FDA says.

The most common adverse reactions associated with Hemgenix included liver enzyme elevations, headache, mild infusion-related reactions, and flu-like symptoms. The notice says patients should be monitored for adverse infusion reactions and liver enzyme elevations in their blood.

The Behring application received priority review and orphan drug and breakthrough therapy designations.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome