Drugs

Support for Hemophilia Gene Therapy Guidance

BioMarin says it commends FDA for providing recommendations for stakeholders developing human gene therapy products to treat hemophilia. The company’s comment says that overall it supports the recommendations in the agency’s draft guidance as a positive step toward providing regulatory clarity for developing these novel products. The comment letter addresses the importance of patient-focused development, traditional approval, and maintaining the regulatory standard for accelerated approval.

In its comment, the American Society of Gene & Cell Therapy says the agency’s recommendations “are generally welcomed and will provide clarity for development of gene therapy products for hemophilia.” The society also provides specific line-by-line comments.

Bayer says the FDA definition of gene therapy includes gene editing, but the draft guidance does not specifically address gene editing. “It would be important to clarify whether any of the components or the combination of the different components of gene editing … would need to be tested in vitro or in nonclinical studies,” the letter 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