The FDA has granted 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.
According to Sangamo Therapeutics:
“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,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Diseases Research Unit. “If FVIII levels are sustained, and patients continue to have no bleeding episodes and remain off factor replacement therapy, we believe that this gene therapy may potentially represent a transformative treatment paradigm for severe hemophilia A.”
FDA Approved RMAT Designations
To date, 30 RMAT designations have been publicly announced. The most recent additions were Krystal Biotech’s RMAT designation for KB103 on June 24, 2019 and Sangamo Therapeutics’ RMAT designation for SB-525 on July 5, 2019.
While the list of publicly announced RMAT’s has risen to 30, the U.S. FDA states it has received 108 total requests for RMAT designations and granted 40. This means approximately 37% of RMAT applications get approved.
The table below presents the cumulative number of RMAT requests that the FDA has received by year.
*Source: U.S. FDA