Biologics

BioMarin Withdrawing Hemophilia A Gene Therapy

After failing to find a buyer, BioMarin has decided to voluntarily withdraw from the market its Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for treating adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5.

Since its 2023 approval, Roctavian failed to achieve commercial success due to low adoption caused by its complex administration, narrow patient eligibility, and limited, unpredictable durability of its gene-therapy effects, industry observers say. Despite a high $2.9 million price tag, patient reluctance to switch from effective, standard chronic treatments and slow, complicated reimbursement processes resulted in poor sales.

Hemophilia A is a rare, genetic condition caused by a mutation in the gene responsible for producing the Factor VIII protein (FVIII), which is necessary for blood clotting, according to the company. The one-time, single-dose infusion is designed to replace the function of the mutated gene, allowing people with severe hemophilia A to produce their own FVIII and thereby limit bleeding episodes.

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