FDA opens review of CAR-T safety after identifying 19 cases of...

FDA opens review of CAR-T safety after identifying 19 cases of post-treatment blood cancer

Nicole DeFeudis

Editor

November 28, 2023 02:54 PM EST

The FDA is reviewing the safety of CAR-T therapies after reports of patients who developed blood cancers following treatment with the products, an inquiry that’s almost certain to raise questions about the class as its developers seek to expand its use.

The agency told Endpoints News that it has received 19 reports since the approval of the first CAR-T therapies in 2017, including 14 from the FDA’s self-reporting adverse event system known as FAERS.

In a press release earlier Tuesday, the agency said that “the overall benefits of these products continue to outweigh their potential risks for their approved uses,” though the agency is currently “evaluating the need for regulatory action.”

Secondary cancers are a known but uncommon risk of many anti-cancer treatments, from chemotherapy to some gene therapies. That includes CAR-Ts, which involve collecting a patient’s own immune cells and engineering them to recognize and attack tumor cells.

Regulators said the risk is applicable to all six CAR-Ts currently on the market, including Bristol Myers Squibb and 2seventy bio’s Abecma (idecabtagene vicleucel); Bristol Myers’ Breyanzi (lisocabtagene maraleucel); Janssen and Legend Biotech’s Carvykti (ciltacabtagene autoleucel); Novartis’ Kymriah (tisagenlecleucel); and Kite Pharma’s Tecartus (brexucabtagene autoleucel) and Yescarta (axicabtagene ciloleucel).

Shares plunged for some CAR-T developers after the FDA announcement, with the smaller biotechs hardest hit.

A surprise from the agency

Stephan Grupp, lead investigator for the US’ first approved CAR-T Kymriah, told Endpoints on Tuesday that the FDA’s announcement “came out of the blue.” Grupp is a University of Pennsylvania professor and a physician at the Children’s Hospital of Philadelphia.

“In the 500 patients that we’ve treated at CHOP with CAR-T, and across all the clinical trials that I’ve been involved in, this has not been a signal at all,” Grupp said.

In response to a question from Endpoints, Gilead said that it had treated 17,700 patients and was confident of the therapy’s safety.

“There is no evidence to date that treatment with Yescarta or Tecartus has a causal role in the development of new malignancies,” a Gilead spokesperson said in an email on Tuesday. “We have a rigorous process in place to continuously monitor for and report adverse events to regulatory authorities.”

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