Drugs

Gene Therapy Mfg. Guidance Coming: Gottlieb

FDA commissioner Scott Gottlieb says the agency will follow its 11/2017 regenerative medicine advanced therapy designation guidance with a similar framework explaining how FDA will address manufacturing issues and the development pathway for gene therapy products. In a 5/22 presentation to the Alliance for Regenerative Medicine annual board meeting, Gottlieb said FDA has more than 500 active investigational NDAs involving gene therapy products, with more than 100 of the applications received last year alone.

“FDA intends to release a suite of draft guidance documents articulating its framework for the manufacturing and clinical development of gene therapy products,” he said. “A lot of this new guidance will be focused on the product-related issues, but these guidances also will provide recommendations for clinical development in certain areas.”

He said the documents will lay out potential accelerated approval endpoints for certain gene therapy products. The first therapeutic area to be focused on will be hemophilia where, he said, factor production may be sufficient in some cases as a surrogate measure of benefit where a gene therapy product can potentially normalize factor production.

“By providing clarity to developers on manufacturing parameters, safety measures, and the pathway toward clinical development,” he said, “FDA hopes to foster even greater innovative development in this field.”

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