The FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation

On February 15, 2019, the FDA released guidance for the development of regenerative medicine therapies targeted to serious conditions and a second guidance on how the FDA will evaluate devices used with regenerative medicine advanced therapies. Draft guidance documents were issue in November 2017 and based on feedback the FDA received, the final documents were issue in February 2019. 

The Regenerative Medicine Advanced Therapy (RMAT) designation was created as part of the 21st Century Cures act. A RMAT is a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product that is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition. The RMAT must also have preliminary evidence that supports the drug has the potential to address an unmet medical need.

The first document describes expedited programs available for companies developing cell-based therapies and gene therapies that would qualify for RMAT status. The second document reviews the process and considerations by the FDA in evaluating devices designed for the recovery, isolation or delivery of RMATs.
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​The Pharmaceutical Pipeline Tracker is currently monitoring four drugs with RMAT designation.

• Ixmyelocel-T is a Phase II stem cell therapy being developed by Vericel for the treatment of heart failure. The drug also has Orphan Drug and Fast Track status.
• VY-AADC is a gene replacement that enables neurons in the brain to express the AADC enzyme that converts levodopa into dopamine. The Phase II drug is being developed by Voyager Therapeutics.
• CD34+ stem cell therapy is a Phase III treatment that is being developed by Caladrius to treat refractory angina
• MPC-150-IM is a Phase III stem cell therapy being developed by Mesoblast for the treatment of moderate to advanced congestive heart failure.