Drugs

Comments on Neurodegenerative Disease FDA Draft Guidance

Novartis says an FDA draft guidance, Human Gene Therapy for Neurodegenerative Diseases, should leverage past learnings from small molecule, biologic, and current gene therapy experiences to develop a platform approach to support regulatory decision-making. The company comment letter says well-characterized common attributes shared across programs can facilitate the use of a common data set without unnecessary redundant studies. It says a science-based platform approach would enable efficiencies needed to bring more transformative therapies to patients.

In its comment, Biogen says that while the draft is a useful overview of high-level recommendations in existing gene therapy guidance, more detailed recommendations are necessary to help guide sponsors as they navigate development. For example, it says, the guidance does not distinguish a rare, rapidly progressing neurological disease from a chronic, slowly progressing disease, although the considerations and rationale may be very different between the two settings. Biogen encourages the agency to develop disease-specific guidance for neurodegenerative diseases that incorporate guidance specific to the development of gene therapy products for those industries. And it supports the use of question-and-answer documents as an alternative approach to inform sponsors of current FDA policy for gene therapies that may not be amenable to the longer lifecycle of guidance development.

Passage Bio makes recommendations on chemistry, manufacturing, and controls, and on considerations for clinical trials.

All three comment letters include detailed, line-by-line technical comments and recommendations.

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