Drugs

Regeneron Plans Garetosmab FDA Submission

Regeneron says it will apply to FDA by the end of 2025 for approval of its garetosmab to treat the ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP). A company statement says the application will be based on the results of the Phase 3 OPTIMA trial that demonstrated that garetosmab prevents over 99% of abnormal bone formation.

The statement says that garetosmab is the only treatment to demonstrate a dramatic reduction in both the number and volume of abnormal bone lesions (heterotopic ossification or HO) in adults with FOP. Regeneron says the trial’s independent data monitoring committee recommended that trial participants receiving a placebo be transitioned to the treatment as soon as possible.

Garetosmab is described as a Velocclumne-derived fully-human monoclonal antibody that binds and neutralizes Activin A, which is involved in the development of heterotopic bone in people with FOP. The company says its Velocclumne technology uses a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies.

FDA has granted garetosmab fast-track designation for the prevention of HO in patients with FOP.

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