With recent news from Sangamo Therapeutics and Krystal Biotech, the count of announced RMAT (“Regenerative Medicine Advanced Therapy”) designations has risen to 30.
Krystal Biotech received an RMAT designation for its gene therapy KB103 on June 24, 2019. KB103 is being studied for its wound closure capacity in patients with severe generalized recessive dystrophic epidermolysis bullosa (“RDEB”). The FDA also granted a RMAT designation for Sangamo Therapeutics’ SB-525, an investigational gene therapy being developed in collaboration with Pfizer to treat severe hemophilia A. SB-525 is the first hemophilia A gene therapy to receive RMAT designation.
As stated by Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Diseases Research Unit, “We are encouraged by the initial clinical data suggesting safety, tolerability, and efficacy of SB-525 and are beginning preparations, including manufacturing, to potentially advance into a registrational study. We are also encouraged by our interactions with regulators and by the FDA’s recent RMAT designation.”
FDA Approved RMATs
Although 30 RMATs have been announced, the U.S. FDA states it has received 108 total requests and granted 40. Therefore, 10 companies are operating in stealth mode with regard to their RMAT designations and approximately 37% ofRMAT applications get approved.
Because RMAT designations allow for faster, more streamlined approvals of regenerative medicine products within the U.S., these companies represent some of the most innovative competitors within the marketplace.
Other regenerative medicine (RM) companies are also emerging every week, with Century Therapeutics emerging from stealth mode on July 1, 2019, with a mission to produce vast amounts of allogeneic, homogeneous iPSC-derived cellular therapeutics.
To keep up with this exponential rate of change, BioInformant maintains the world’s only searchable, sortable database of regenerative medicine companies worldwide that is continuously updated on a daily basis.