WASHINGTON, D.C., March 18, 2026 — The U.S. Food and Drug Administration has introduced a notable policy shift that could benefit developers of cell and gene therapies, announcing it will now consider granting Regenerative Medicine Advanced Therapy (RMAT) designation even when a product is under clinical hold.
The RMAT designation, overseen by the FDA’s Center for Biologics Evaluation and Research, is designed to accelerate the development of regenerative medicines for serious or life-threatening conditions. It enables early and frequent interaction with regulators, along with benefits similar to fast track and breakthrough therapy designations, including potential for expedited approval pathways.
Previously, FDA guidance explicitly stated that RMAT designation would not be granted if an investigational new drug (IND) application was on clinical hold during review. However, under updated standard operating procedures effective March 12, 2026, the agency will now evaluate such requests on a case-by-case basis.
According to the revised policy, the FDA will assess whether the issues leading to a clinical hold prevent determination of the therapy’s potential to address unmet medical needs. If not, the therapy may still qualify for RMAT designation despite the hold status.
The agency has not publicly detailed the rationale behind the change. However, the update comes amid increasing scrutiny over the FDA’s use of clinical holds in the rapidly evolving cell and gene therapy sector.
In 2025, the FDA received a record 91 RMAT designation requests, granting 50 and denying 22. At the same time, clinical holds have become more common, reflecting both the complexity of advanced therapies and heightened regulatory caution.
Concerns over regulatory practices have also been raised by policymakers. Bill Cassidy, chairman of the Senate HELP Committee, recently urged the FDA to apply clinical holds more judiciously. He cited industry feedback describing opaque communication, late-stage regulatory interventions, and limited transparency in decision-making.
Despite these challenges, historical data suggest that most clinical holds are temporary. A 2023 analysis found that approximately 80% of holds were eventually lifted, typically within about six months.
The FDA’s updated RMAT policy may provide greater flexibility for smaller biotech companies, which often face significant delays and financial strain when trials are paused. By allowing earlier regulatory engagement—even during a hold—the agency could help sponsors address issues more efficiently and maintain development momentum.
Overall, the change signals a more adaptive regulatory approach as the FDA continues to balance innovation with safety in the growing field of regenerative medicine.
