Biologics

FDA Confident Zolgensma Should Stay on Market

FDA says it is confident that AveXis’ Zlogensma, a gene therapy product intended to treat children less than two years old with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 gene, that was approved by the agency 5/24, should remain on the market despite data manipulation concerns raised by the manufacturer after approval. An agency statement says FDA was informed 6/28 about a data manipulation issue that affects the accuracy of certain data from product testing performed in animals submitted in the BLA reviewed by FDA.

The statement says the agency is carefully assessing the situation and “remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was included in the marketing application. This product testing data was used by the manufacturer to support the development of its production process for the product. These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program.”

FDA says the totality of the evidence demonstrating the product’s effectiveness and its safety profile continues to provide compelling evidence supporting an overall favorable benefit-risk profile.

The statement acknowledges that the manufacturer became aware of the data manipulation before FDA approved the product and did not inform the agency until after the approval. It says the agency will act against the company, if appropriate, and could impose civil or criminal penalties.

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