The U.S. Food and Drug Administration (FDA) announced on Friday that it has eliminated the Risk Evaluation and Mitigation Strategies (REMS) for currently approved BCMA- and CD19-directed autologous chimeric antigen receptor CAR T cell immunotherapies.
These products are gene therapies currently approved to treat blood cancers, such as multiple myeloma and certain types of leukemia and lymphoma.
“The FDA has taken the bold step to remove the Risk Evaluation and Mitigation Strategy requirement from giving CAR T therapies. REMS is a useful safety system, but reevaluation over time helps inform whether a REMS is still needed to ensure that the benefits of a product outweigh its risks,” said FDA Vinay Prasad, Chief Medical and Scientific Officer and Director, Center for Biologics Evaluation and Research.
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“Eliminating the REMS that is no longer needed also expedites the delivery of potentially curative treatments to patients and reduces the burden on providers,” he added.
A REMS is a safety program that the FDA can require for certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks.
The FDA determined that the approved REMS for the following products should be eliminated because a REMS is no longer necessary to ensure that the benefits of the autologous CAR T cell immunotherapies outweigh their risks.
The products affected by this change include Bristol Myers Squibb & Co.’s BMY Breyanzi (lisocabtagene maraleucel) and Abecma (idecabtagene vicleucel), Johnson & Johnson’s JNJ Carvykti (ciltacabtagene autoleucel), Novartis AG’s NVS Kymriah (tisagenlecleucel), and Gilead Science Inc.’s GILD Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel).
The elimination of REMS for the above products removes the requirements that hospitals and their associated clinics that dispense products must be specially certified and have on-site, immediate access to tocilizumab.
“Continuous monitoring and assessment of the safety of all biological products, including the CAR T cell immunotherapies, is an FDA priority and we remain committed to informing the public when we learn new information about these products,” the FDA said in a press release.
As per a safety statement on Thursday, the regulator also reduced its requirement that patients remain near the facility where they received treatment to two weeks. The agency also removed its advice to patients to avoid driving for two weeks following product administration.
Commenting on the development and accessibility of CAR T-cell therapies, William Blair said, “Overall, we view the removal of the REMS program for CAR-T cell therapies as a positive development for the space, as it supports the notion that the FDA is working to minimize red tape and increase access to potentially curative treatments for patients. We also believe the removal of the REMS requirements could increase access and use of these therapies in the community settings.”
Analyst Sami Corwin said the update also suggests there will likely be no REMS requirements for CD19 and BCMA autologous CAR-T cell therapies approved in the future, such as Arcellx, Inc’s ACLX anito-cel.
Moreover, Corwin highlighted the positive implications for companies developing CAR T-cell therapies for autoimmune diseases, such as Autolus Therapeutics plc AUTL, Bristol-Myers, and Cabaletta Bio, Inc CABA, “given that the safety profile is more tolerable than in oncology.”
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