October 28, 2020

The Niche

Knoepfler lab stem cell blog

RMAT List (46)

FDA RMAT

Here is a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT.

FDA RMAT
Screenshot from FDA Voice page on RMAT going live

I’m assuming for the purposes of this resource page that all self-reports are accurate. Note that the links embedded into the firm names are to relevant press releases. A special thanks to some folks at ARM for helping me keep the list up to date.

It was updated as of October 6, 2020 with 46 RMAT total. Note that the FDA has recently updated it’s RMAT totals to indicate that it has approved 52 RMATs as of mid-2020 so about 8 are not in the public domain.

Here’s the list.

  1. Abeona Therapeutics (EB-101 Gene Therapy in Epidermolysis Bullosa)
  2. Abeona Therapeutics (ABO-102 Gene Therapy in MPS IIIA)
  3. AdaptImmune (ADP‑A2M4 for the Treatment of Synovial Sarcoma)
  4. AlloVir (Viralym-M for hemorrhagic cystitis)
  5. Asterias/BioTime (AST-OPC1, spinal cord injury)
  6. Athersys (MultiStem)
  7. Athersys (MuliStem for ARDS)
  8. Audentes (AT132 for the Treatment of X-Linked Myotubular Myopathy)
  9. AxoGen (Avance for nerve injuries)
  10. BlueBird Bio (Lentiglobin in SCID)
  11. Bristol-Myers Squibb (Liso-cel, CAR-T cell therapy for large B-cell lymphoma)
  12. Caladrius (CD34+ cell therapy for angina)
  13. Capricor (CAP1002, Duchenne Muscular Dystrophy)
  14. CARsgen (CT053 CAR-T therapy for Multiple Myeloma)
  15. Cellerant Therapeutics, Inc/Fibrocell. (Romyelocel-L to Prevent Infections During Neutropenia)
  16. Enzyvant (DiGeorge syndrome)
  17. ExCellThera (ECT-001 for blood cancers
  18. Fibrocell (FCX-007 Gene Therapy for the Treatment of RDEB)
  19. Fortress Biotech/Cellvation (CEVA101, traumatic brain injury)
  20. Helixmith (VM202 is a DNA based therapy for Diabetic Peripheral Neuropathy)
  21. Humacyte (Vascular Access for Hemodialysis)
  22. Iovance (Lifileucel for metastatic melanoma)
  23. jCyte (Retinitis Pigmentosa)
  24. Immunicum AB (Ilixadencel in Kidney Cancer)
  25. Juno/Celgene (JCAR017; CAR-T)
  26. Kiadis Pharma (ATIR101, blood disorders)
  27. Krystal Biotech (Gene therapy KB103 for skin disease)
  28. Medeor Therapeutics Transplant Immune Tolerance 
  29. Magenta (MGTA-456 for Inherited Metabolic Disorders)
  30. Mallinckrodt/Stratatech’s Stratagraft
  31. Mesoblast (Heart Failure, mesenchymal precursor cell therapy)
  32. MiMedx Group (AmnioFix for osteoarthritis)
  33. Mustang Bio/St. Judes (MB-107 lentiviral gene therapy for X-linked Severe Combined Immunodeficiency (XSCID))
  34. NightStar Therapeutics (NSR-REP1 for choroideremia)
  35. Novartis (Kymriah for follicular lymphoma)
  36. Orchard Therapeutics (OTL-103 for the Treatment of Wiskott-Aldrich Syndrome)
  37. Poseida Therapeutics (P-BCMA-101 for multiple myeloma)
  38. Rocket Pharma (RP-L102 for Fanconi Anemia)
  39. SanBio (SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury)
  40. Sangamo Therapeutics (SB-525 for Hemophilia A.)
  41. Talaris (FCR-001 for prevention of renal transplantation rejection)
  42. Tessa (CAR-T for Hodgkin Lymphoma)
  43. TissueTech (cryopreserved human umbilical cord investigational biologic product TTAX02 for spina bifida in-utero.)
  44. Vertex/CRISPR Therapeutics (CTX001 for the Treatment of Severe Hemoglobinopathies)
  45. Vericel (Ixmyelocel-T for cardiovascular disease)
  46. Voyager Therapeutics (VY-AADC for the Treatment of Parkinson’s Disease)