This post includes my updated list of firms/products that have received FDA regenerative medicine advanced therapy designation or RMAT, which are in the public domain.
The numbers really jumped in the last month.
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 or other reference material.
A special thanks to some folks at ARM for helping me keep the list up to date.
You can stay up to date on the running total of RMATs in the public domain here.
You can see at right a picture of CBER Director Peter Marks from the original FDA blog post when RMATs first went live just about 2.5 years ago.
I wonder how many of these regenerative therapies will get full FDA approval in the end? My hope is quite a few, which would justify the RMAT program. I don’t believe it’ll be zero. This is one of the most important questions for the stem cell field.
Here’s the list updated as of September 24, 2019 with a remarkable 36 total:
- Abeona Therapeutics (EB-101 Gene Therapy in Epidermolysis Bullosa)
- Abeona Therapeutics (ABO-102 Gene Therapy in MPS IIIA)
- AlloVir (Viralym-M for hemorrhagic cystitis)
- Asterias/BioTime (AST-OPC1, spinal cord injury)
- Athersys (MultiStem)
- Audentes (AT132 for the Treatment of X-Linked Myotubular Myopathy)
- AxoGen (Avance for nerve injuries)
- BlueBird Bio (Lentiglobin in SCID)
- Caladrius (CD34+ cell therapy for angina)
- Capricor (CAP1002, Duchenne Muscular Dystrophy)
- Cellerant Therapeutics, Inc/Fibrocell. (Romyelocel-L to Prevent Infections During Neutropenia)
- Enzyvant (DiGeorge syndrome)
- ExCellThera (ECT-001 for blood cancers
- Fibrocell (FCX-007 Gene Therapy for the Treatment of RDEB)
- Fortress Biotech/Cellvation (CEVA101, traumatic brain injury)
- Humacyte (Vascular Access for Hemodialysis)
- Iovance (Lifileucel for metastatic melanoma)
- jCyte (Retinitis Pigmentosa)
- Juno/Celgene (JCAR017; CAR-T)
- Kiadis Pharma (ATIR101, blood disorders)
- Krystal Biotech (Gene therapy KB103 for skin disease)
- Magenta (MGTA-456 for Inherited Metabolic Disorders)
- Mallinckrodt/Stratatech’s Stratagraft
- Mesoblast (Heart Failure, mesenchymal precursor cell therapy)
- MiMedx Group (AmnioFix for osteoarthritis)
- Mustang Bio/St. Judes (MB-107 lentiviral gene therapy for X-linked Severe Combined Immunodeficiency (XSCID))
- NightStar Therapeutics (NSR-REP1 for choroideremia)
- Orchard Therapeutics (OTL-103 for the Treatment of Wiskott-Aldrich Syndrome)
- Poseida Therapeutics (P-BCMA-101 for multiple myeloma)
- Rocket Pharma (RP-L102 for Fanconi Anemia)
- SanBio (SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury)
- Sangamo Therapeutics (SB-525 for Hemophilia A.)
- Talaris (FCR-001 for prevention of renal transplantation rejection)
- Vericel (Ixmyelocel-T for cardiovascular disease)
- ViroMed (VM202 is a DNA based therapy for ALS)
- Voyager Therapeutics (VY-AADC for the Treatment of Parkinson’s Disease)
Peter Marks is amazing – he used to be on the Yale faculty and is one of the most hard-working and productive people I’ve ever met. We are very lucky he is head of CBER at the FDA!
Definitely!