Drugs

CMC Human Gene Therapy Guidance ‘Inadequate’: PhRMA

Pharmaceutical Research and Manufacturers of America (PhRMA) says an FDA draft guidance on chemistry, manufacturing, and control (CMC) information for human gene therapy INDs is a positive step toward providing sponsors with more guidance and clarity, but “does not adequately adopt and identify risk-based and phase-appropriate recommendations that are tailored to IND submissions for these therapies.” The PhRMA letter gives comments and recommendations on guidance scope and structure, risk-based CMC approaches, and agency processes and policies. It also includes line-by-line editing recommendations.

The American Society of Gene and Cell Therapy says the draft’s recommendations “are generally welcomed and will provide clarity on CMC information for IND applications for human gene therapy.” The letter also contains line-by-line recommendations.

Pfizer comments that it is “generally supportive of the guidance” and suggests that recommendations on devices should be discussed in one specific section rather than dispersed across the guidance. The company also says it would be helpful to have more detail on a phase-driven approach to submitting information.

The Innovative Genomics Institute asks FDA to provide more specific and tailored guidance and recommendations for drug substances and products created by gene editing technologies, particularly those therapeutics that are created ex vivo using editing reagents that are not intended to persist.

Finally, the Parenteral Drug Association says the draft guidance “varies between the material presented for INDs and GMP requirements.” It recommends that either it be reorganized or the IND-relevant information be removed from IND quality practices and placed in a separate guidance.

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