Regenerative medicine advanced therapy

Regenerative Medicine Advanced Therapy (RMAT) is a designation given by the Food and Drug Administration to drug candidates intended to treat serious or life-threatening conditions under the 21st Century Cures Act.[1] A RMAT designation allows for accelerated approval based surrogate or intermediate endpoints.[2]

RMAT goes beyond breakthrough therapy features by allowing for accelerated approval of drugs based on surrogate endpoints. A surrogate endpoint is a biomarker that substitutes for a direct endpoint, such as clinical benefit.[3]

  1. ^ "Regenerative Medicine Advanced Therapy Designation". Food and Drug Administration. Archived from the original on 2 August 2021. Retrieved 25 June 2021.
  2. ^ Vaggelas, Annegret; Seimetz, Diane (2019-05-01). "Expediting Drug Development: FDA's New Regenerative Medicine Advanced Therapy Designation". Therapeutic Innovation & Regulatory Science. 53 (3): 364–373. doi:10.1177/2168479018779373. ISSN 2168-4804. PMID 29895180. S2CID 48355647.
  3. ^ Aronson, J K (2005). "Biomarkers and surrogate endpoints". British Journal of Clinical Pharmacology. 59 (5): 491–494. doi:10.1111/j.1365-2125.2005.02435.x. PMC 1884846. PMID 15842546.
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