Updated FDA granted RMAT List (85)

Today’s post includes a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT.

Sometimes people call these “r mat” with a space.

As of December 12, 2023 there are 85 RMATs in the public domain.

I also include FDA data on RMAT submissions via an FDA RMAT resource page.

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.

RMAT, FDA data
FDA table on data related to their review of regenerative medicine advanced therapy designation or RMAT applications. Screenshot from FDA site with data from December 12, 2023.

Note that the FDA has recently updated its RMAT totals to indicate that it has granted a total of 92 RMATs so 7 are not in the public domain that I could find. You can see the interesting table above from the FDA about the number of total applications, those granted, and those denied or withdrawn. In every year more RMATs were denied or withdrawn than granted. I wonder how many applications were from stem cell clinics? The agency continues to deny a sizable number of applications.

Here’s my list of the 85 granted RMATs that I could find in the public domain.

  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. AdaptImmune (ADP-A2M4CD8 for the treatment of patients with platinum resistant ovarian cancer)
  5. Allogene Therapeutics (ALLO-715, an AlloCAR T Cell Therapy for Relapsed/Refractory Multiple Myeloma
  6. Allogene Therapeutics (ALLO-501A for Large B Cell Lymphoma)
  7. AlloVir (Viralym-M for hemorrhagic cystitis)
  8. AlloVir (Viralym-M for adenovirus infections after stem cell transplants.
  9. AlloVir (Viralym-M for viral infections more generally after transplants. This seems to overlap with the previously listed one).
  10. Angiocrine (AB205 Cell therapy for organ vascular niche injuries for the prevention of severe toxicities in lymphoma patients)
  11. Arcellx (CART-ddBCMA for refractory multiple myeloma)
  12. Athersys (MultiStem)
  13. Athersys (MuliStem for ARDS)
  14. Atsena Therapeutics  (ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)
  15. Audentes (AT132 for the Treatment of X-Linked Myotubular Myopathy)
  16. Autolus (obe-cel, for the treatment of adult B-ALL)
  17. AxoGen (Avance for nerve injuries)
  18. BioMarin (Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A)
  19. BlueBird Bio (Lentiglobin in SCID)
  20. BlueBird Bio (lovotibeglogene autotemcel (lovo-cel) for certain cases of sickle cell)
  21. Bristol-Myers Squibb (Liso-cel, CAR-T cell therapy for large B-cell lymphoma)
  22. Caladrius (CD34+ cell therapy for angina)
  23. Capricor (CAP1002, Duchenne Muscular Dystrophy)
  24. Caribou Bio (CB-010, an Allogeneic Anti-CD19 CAR-T Cell Therapy for LBCL)
  25. CARsgen (Zevor-cel or CT053 CAR-T therapy for Multiple Myeloma)
  26. CARsgen (CT041 CAR-T therapy for specific GI cancers)
  27. Cellerant Therapeutics, Inc/Fibrocell. (Romyelocel-L to Prevent Infections During Neutropenia)
  28. Cellular Biomedicine Group or CBMG. C-CAR039 for relapsed or refractory diffuse large B cell lymphoma)
  29. Cook MyoSite (AMDC-USR for Urinary Sphincter Repair)
  30. Cook MyoSite (AMDC-USR for Urinary Sphincter Repair)
  31. CRISPR Therapeutics (CTX001 for hemoglobinopathies. This is with Vertex)
  32. CRISPR Therapeutics (CTX110 for specific B cell tumors)
  33. CRISPR Therapeutics (CTX130 for cutaneous T cell lymphomas)
  34. Direct Biologics (ExoFlo for COVID-19). This one is highly controversial in my view. There was political pressure on then Commissioner Steve Hahn to move this forward for Direct Biologics. My review of the key ExoFlo Phase I trial paper found many red flags too.
  35. DiscGenics (IDCT or rebonuputemcel for disc repair)
  36. Editas (EDIT-301 for severe sickle cell disease).
  37. Enzyvant (DiGeorge syndrome)
  38. ExCellThera (ECT-001 for blood cancers)
  39. Fate Therapeutics (FT516 for Relapsed / Refractory DLBCL)
  40. Fibrocell (FCX-007 Gene Therapy for the Treatment of RDEB)
  41. Fortress Biotech/Cellvation (CEVA101, traumatic brain injury)
  42. Helixmith (VM202 is a DNA based therapy for Diabetic Peripheral Neuropathy)
  43. Humacyte (Human Acellular Vessel (HAV) for urgent arterial repair following extremity vascular trauma.)
  44. Humacyte (Vascular Access for Hemodialysis)
  45. IASO Biotherapeutics (BCMA CAR-T CT103A for relapsed/refractory multiple myeloma)
  46. Immatics (IMA203 GEN1 for multiple PRAME-expressing cancers, including cutaneous and uveal melanoma).
  47. Immunicum AB (Ilixadencel in Kidney Cancer)
  48. Intellia Therapeutics (NTLA-2002 for angioedema)
  49. Iovance (Lifileucel for metastatic melanoma)
  50. jCyte (Retinitis Pigmentosa)
  51. Juno/Celgene (JCAR017; CAR-T)
  52. Kiadis Pharma (ATIR101, blood disorders)
  53. Krystal Biotech (Gene therapy KB103 for skin disease)
  54. Lineage Cell Therapeutics (OPC1, spinal cord injury)
  55. Magenta (MGTA-456 for Inherited Metabolic Disorders)
  56. Mallinckrodt/Stratatech’s Stratagraft
  57. Medeor Therapeutics (MDR-101 for prevention of kidney transplant rejection)
  58. Mesoblast (Heart Failure, mesenchymal precursor cell therapy Rexlemestrocel-L)
  59. Mesoblast (Back pain, Rexlemestrocel-L)
  60. MiMedx Group (AmnioFix for osteoarthritis)
  61. Mustang Bio/St. Judes (MB-107 lentiviral gene therapy for X-linked Severe Combined Immunodeficiency (XSCID))
  62. NightStar Therapeutics (NSR-REP1 for choroideremia)
  63. Novartis (Kymriah for follicular lymphoma)
  64. Ocugen (NeoCart for generating new cartilage for OA)
  65. Orca Bio (Orca-T (NCT04013685 for patients with blood cancers eligible for HSCT)
  66. Orchard Therapeutics (OTL-103 for the Treatment of Wiskott-Aldrich Syndrome)
  67. Orchard Therapeutics (OTL-200 for Metachromatic Leukodystrophy)
  68. Organogenesis (ReNu candidate for osteoarthritis of the knee)
  69. PolarityTE (SkinTE for cutaneous ulcers)
  70. Poseida Therapeutics (P-BCMA-101 for multiple myeloma)
  71. ProKidney (REACT for serious kidney disease)
  72. Regenxbio (RGX-121 for Hunter syndrome). This is a gene therapy.
  73. Rocket Pharmaceuticals (RP-L102 for Fanconi Anemia)
  74. Rocket Pharmaceuticals (RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I)
  75. Rocket Pharmaceuticals (RP-L301 for pyruvate kinase deficiency)
  76. Rocket Pharmaceuticals (RP-A501 for Danon Disease)
  77. SanBio (SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury)
  78. Sangamo Therapeutics (SB-525 for Hemophilia A.)
  79. Takeda (Darvadstrocel for Perianal fistulas)
  80. Talaris (FCR-001 for prevention of renal transplantation rejection)
  81. Tessa (CAR-T for Hodgkin Lymphoma)
  82. TissueTech (cryopreserved human umbilical cord investigational biologic product TTAX02 for spina bifida in-utero.)
  83. UCSF (Artemis-SCID treatment: I don’t see a product name here).
  84. Vericel (Ixmyelocel-T for cardiovascular disease)
  85. Voyager Therapeutics (VY-AADC for the Treatment of Parkinson’s Disease)

References

  1. FDA RMAT page, current as of December 12, 2023.

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