Drugs

FDA Grants, Denies Catalyst Amifampridine Petition

FDA has granted in part and denied in part a Catalyst Pharmaceuticals 7/8/21 petition asking the agency not to consider any NDA to use amifampridine to treat congenital myasthenic syndromes (CMS) approvable based on a mechanistic argument of efficacy and to require the NDA to include well-controlled, randomized clinical trials as evidence of efficacy and safety. The petition also asked FDA to issue guidance with several of the company’s recommendations.

The FDA letter says cases of CMS vary in their clinical features and their response to treatment. “Because of the heterogeneity in the phenotypes and genotypes of congenital myasthenia,” it says, “FDA does not currently have evidence that a mechanistic rationale alone would be sufficient for the approval of a drug to treat CMS. We decline, however, to state that a mechanistic argument of efficacy could never provide evidentiary support for the treatment of CMS of genetic origin with amifampridine. The agency will consider any NDA for amifampridine for treatment of CMS on a case-by-case basis.”

FDA also declined to issue guidance at this time with recommendations on how to structure clinical trials studying genetic subtypes of CMS. It says it will continue to provide advice to sponsors on a case-by-case basis.

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