Biologics

New FDA Flexibility in Cell and Gene Therapy Approvals

A 5/5 FDA guidance on chemistry, manufacturing, and controls flexibilities for developing human cellular and gene therapy (CGT) products is changing the landscape of regulatory approval for such products. A BioProcess International online column analyzing the guidance, which is being implemented immediately, says FDA has abandoned its long-standing requirement for three commercial validation batches for therapies that, due to small patient populations or individualized manufacturing, might be produced in only a handful of lots over their lifetimes.

The guidance discusses 13 specific agency flexibility provisions arranged in four distinct areas:

clinical development;
process validation;
commercial specifications; and
additional provisions.

“Industry experts have welcomed the clarity provided by the final guidance,” the post says. “FDA acknowledged that it can be difficult for sponsors to create CGT products using traditional product development strategies…. The agency states in the guidance, ‘Consistent with the statutory and regulatory requirements for biological products, FDA uses a flexible approach to ensuring applicable CMC requirements are met for CGT products’ It says such an approach ‘serves to help expedite development, review, and patient access to safe and effective CGT products to treat serious or life-threatening conditions [with] significant unmet medical needs.’”

The column makes clear that although the guidance eliminates procedural rigidity, it explicitly introduces no concessions to the foundational pillars of FDA quality compliance. “Regulators have drawn a firm line,” it says, “flexibility in batch quantity does not equal flexibility in data integrity, patient safety, or product purity.”

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