Biologics

Cell/Gene Therapy Guide on Common Questions

FDA has posted a draft guidance entitled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products.” The document is intended to provide industry with answers to frequently asked questions (FAQs) and commonly faced issues that arise during cell/gene therapy development. The FAQs cover multiple disciplines, including regulatory review, chemistry, manufacturing, and controls, pharmacology/toxicology., clinical, and clinical pharmacology.

For example, under clinical endpoints for traditional approval, the agency recommends that these “directly reflect a meaningful clinical benefit (i.e., how study participants feel or function, or how long they survive) or validated surrogate endpoints (i.e., those that have been shown to predict a specific clinical benefit).” For accelerated approval, the agency says the endpoint should produce evidence of a demonstrated effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit or on an intermediate clinical endpoint (an endpoint that can be measured earlier than irreversible morbidity or mortality).

Regarding safety, FDA says that because many cell/gene therapy products are administered once, close subject monitoring “immediately following product administration is critical to capture early safety signals. This means that during and immediately following product administration, there should be intensive safety monitoring with frequent monitoring of vital signs, physical examinations, laboratory studies, radiologic evaluations, and other relevant studies as warranted. During the subsequent weeks and months, subjects should be monitored frequently for assessment of emerging safety signals via clinical evaluation and ancillary testing.”

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