Biologics

FDA Raises Approval Standards for CAR-T Cancer Therapies

FDA is signaling a major policy shift for cancer CAR T-cell therapies, moving away from reliance on single-arm studies toward randomized controlled trials (RCTs) that demonstrate superiority over existing treatments. Writing in a new JAMA Perspective piece, CBER director Vinay Prasad and other Center officials say that many new CAR-T therapies will now be expected to show that their products outperform (superiority) standard-of-care treatments. This marks a departure from the approach used since the agency approved Novartis’ Kymriah in 2017, which largely relied on single-arm trials.

Writing in JAMA, Prasad et al say the agency’s goal is to maintain high evidentiary standards while using regulatory flexibilities when appropriate. They recommend that initial approvals for CAR-Ts be based on RCTs with clinically meaningful endpoints such as overall survival or acceptable time-to-event measures.

“Evidence should generally demonstrate superiority of the investigational CAR T-cell therapy compared with control,” the authors write. Plans to seek approval based on equivalence or noninferiority will require FDA discussion and justification.

The choice of control groups is critical, their guidance notes. Sponsors must carefully assess standard-of-care options and ethical considerations to ensure trial results reliably reflect the therapy’s effects rather than other factors. Single-arm trials may still be acceptable in rare cancers or in heavily pretreated populations where RCTs are not feasible. Accelerated approvals may still be granted based on high, durable response rates in these contexts, but traditional approval will require evidence from RCTs showing clinical benefit.

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