The FDA has just made some key regulatory changes to make cancer cell therapy more accessible
When the automotive Therapies first came to the market, they made warnings and monitoring requirements to protect patients – for good reason. Incorporating the patient's own immune cells into targeted cancer fighters brings the risk of excessive immune response and neurotoxicity. Patients are asked to stay near the treatment center to monitor these risks. They also temporarily banned driving.
As clinicians gain experience in automotive T therapy, they also gain experience in managing their complications. The FDA recognizes that regulators are now modifying the safety requirements for the scope of these cellular therapies. Analysts say these changes are a step to make these treatments more accessible to more patients.
The security risks of CAR T are marked with a black box warning on its label. These warnings will not disappear. However, approval of these therapies also includes requirements for Risk Assessment and Mitigation Strategies (REMS), a safety information program that provides serious safety risks to clinicians and patients. The FDA said late Thursday that it has eliminated REMS requirements for the T-Therapies class. This procedure also eliminates the requirement for these cell therapies to be managed at specially certified sites. These requirements allocate these therapies to specialized hospitals, such as academic medical centers, which means many patients need to travel to treatment.
“The FDA expects REMS elimination and these tag updates will help improve opportunities for these products, especially for patients living in rural areas, while ensuring safe and effective management of patients who need them,” the agency said.
The changes included clinician education and training materials a year after the FDA eliminated the requirement for cell therapy REM. The agency also removed the requirement to report adverse events, suggesting excessive immune response or neurotoxicity, saying that the system already in place is in place for post-market reporting of adverse events is sufficient.
There are currently seven approved T-Therapies for blood cancer. Deleting REMS requirements affects six of them: Abecma and Breyanzi of Bristol Myers Squibb (BMS); Kymriah of Novartis; Carvykti of Johnson & Johnson and Legend Biotech; and Jescata and tecartus of Gilead Sciences. The seventh FDA-approved CAR T-Therapy is Aucatzyl, an automated therapeutic cell therapy designed to improve safety. Last November, Aucatzyl's FDA approval made it the first automotive T-handling without REMS.
In addition to eliminating REMS, the FDA has reduced the requirement for patients to be around the treatment center for four weeks, thus reducing it to two weeks. Previous guidelines also advise against driving or using heavy machinery for at least eight weeks, which will prevent many patients from returning to work. After infusion of cell therapy, the FDA reduces the limit to two weeks.
BMS said it will work closely with more than 150 currently approved treatment centers to manage the Breyanzi and Abecma removal of REMS programs. The company also aims to add community cancer centers nationwide to bring these therapies closer to patients, thus reducing travel time and patient duration must be away from home and work.
Clinicians told the company that previous requests were a significant burden that hindered community referrals and patients’ decision to accept CAR T, said in a note sent to investors. Clinicians added that as automotive T-drive treatments are transferred to outpatient treatments, treatment centers are now overcapacity and can absorb the increased demand for these therapies. Graybosch said the absorption of automotive T-Therapies remains “stubbornly stagnant” with market penetration of about 20%. Relaxing the limitations surrounding using these therapies should help change that.
“We believe that the CAR-T burden for patients and caregivers will drive the expansion of the U.S. cell therapy market due to the FDA's new monitoring and driving requirements,” Graybosch said.
Analysts for William Blair noted that these changes suggest that cancer cell therapies that are still under development, such as Arcellx’s multiple myeloma TT-treatment Anitocabtagene Autoleucel, which is being developed in collaboration with Gilead Sciences and may not require REMS. The company also believes that the change is a positive sign for automated immune disease therapy developers, including automated, BMS and Cabaletta Bio.
Images shared by Flickr users through creative license
