Drugs

Enzyme Replacement Therapy Guidance

FDA has published a guidance, Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment, to help sponsors develop and conduct nonclinical studies during development of investigational enzyme replacement therapy (ERT) products. Specifically, the document describes the agency’s current thinking about the substance and scope of nonclinical information needed to support start of clinical trials, ongoing clinical development, and marketing approval for investigational ERT products.

The guidance is intended as an adjunct to three International Council on Harmonization guidances that give general recommendations about the nonclinical safety studies of traditional small molecule and biotechnology-derived pharmaceuticals that support human clinical trials, as well as marketing authorization for pharmaceuticals.

The FDA guidance recommendations apply to ERT products indicated for lysosomal storage diseases and other diseases caused by inborn errors of metabolism. It does not apply to the development of pancreatic enzyme products.

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