Drugs

FDA OKs Regeneron Therapy for Fibrodysplasia Ossificans Progressiva

FDA has approved Regneron’s Pasatru (garetosmab-grts) for reducing new heterotopic ossification (bone formation outside the skeleton) and reduce clinician-assessed disease flare-ups in adults with fibrodysplasia ossificans progressiva (FOP), a rare genetic disease caused by a mutation in activin A receptor-type 1, which controls new bone growth. “As a result, connective tissues such as muscle, tendons and ligaments gradually turn into bone, causing limited movement, deformities, severe disability, and early death,” FDA says in a release.

Pasatru is an antibody that blocks activation of the abnormal activin A receptor-type 1.It was approved based on data from a randomized, double-blind, placebo-controlled clinical study of 63 adults with FOP. “Patients received Pasatru 3 mg/kg, Pasatru 10 mg/kg, or placebo, each given by IV infusion every four weeks for 56 weeks,” FDA says. “After the initial 56 weeks, 61 patients continued in an extended follow-up phase on the same treatment.”

The agency says that both doses of Pasatru significantly reduced new bone growth compared to placebo (2 new lesions among 23 patients receiving the 10 mg/kg dose and 1 new lesion among 19 patients receiving the 3 mg/kg dose, compared to 19 new lesions among 21 patients receiving placebo). “The number of clinician-assessed disease flare ups over the 56 weeks were 9 with the 10 mg/kg dose, 53 with the 3 mg/kg dose and 66 with placebo,” it says.

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