The U.S. Food and Drug Administration (FDA) announced on Friday that it has removed the Risk Evaluation and Mitigation Strategies (REMS) requirements for all currently approved CAR-T cell immunotherapies. Regulators traditionally require the REMS safety program to ensure that a drug’s benefits outweigh its risks by addressing serious safety concerns.
Safety Measures Shift to Product Labeling
According to the FDA, safety risks associated with CAR-T therapies—such as cytokine release syndrome and neurological toxicities—can now be effectively communicated through existing product labeling. These include boxed warnings and medication guides, which are already in place for these treatments. As a result, the FDA no longer deems the REMS programs necessary.
Leading CAR-T Therapies Affected
The decision impacts several major CAR-T therapies, including:
- Breyanzi and Abecma by Bristol-Myers Squibb and its partner 2seventy bio
- Carvykti by Johnson & Johnson’s Janssen and Legend Biotech
- Kymriah by Novartis
- Yescarta and Tecartus by Gilead Sciences’ Kite Pharma
Following the announcement, Gilead confirmed updates to the labels of Yescarta and Tecartus, in line with the FDA’s new directive. The company welcomed the decision, stating it would help reduce the burden on healthcare providers and patients, while improving access to potentially curative therapies.
CAR-T: A Revolutionary but Complex Therapy
CAR-T cell therapy is a personalized gene therapy that involves collecting a patient’s T-cells (a type of white blood cell), modifying them to target cancer, and re-infusing them into the patient. These therapies are approved to treat blood cancers such as multiple myeloma, leukemia, and lymphoma.
Regulatory Caution Remains
As reported by Reuters, despite the regulatory shift, the FDA continues to monitor the safety of CAR-T therapies closely. In January 2024, the agency required manufacturers to add serious warnings to CAR-T product labels after receiving reports of T-cell malignancies, including secondary leukemia and lymphoma. These rare but serious events could lead to hospitalization or death, prompting continued vigilance.
Industry Response
Lynelle Hoch, President of the Cell Therapy Organization at Bristol-Myers Squibb, emphasized the importance of challenging existing barriers:
“CAR T cell therapy is a transformational, potentially life-saving option for patients living with blood cancers. We are working to challenge current practices, assumptions, and barriers that limit access.”
With this move, the FDA aims to simplify access to CAR-T therapies while still ensuring patient safety through enhanced product labeling and continuous post-market surveillance.