Biologics

2 FDA Gene Therapy Guidances Out

FDA has published two guidances relating to gene therapy products. The first, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, gives the agency’s current thinking on determining the sameness of these products for orphan drug designation and exclusivity. It is intended to assist stakeholders in developing gene therapies for rare diseases. The document focuses specifically on factors FDA generally intends to consider when determining sameness and does not address sameness determinations for other product types. The guidance finalizes a 1/2020 draft.

The second document, Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial, is a draft that provides recommendations to sponsors interested in studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial for a single disease. The agency says sponsors have expressed interest in gathering preliminary evidence of safety and activity using multiple versions of a cellular or gene therapy product in a single clinical trial. In the guidance, FDA gives recommendations for studies evaluating multiple versions of a cellular or gene therapy product, including how to organize and structure the INDs, submit new information, and report adverse events.

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