On September 4, 2019, Magenta Therapeutics announced it had received an FDA Regenerative Medicine Advance Therapy (RMAT) designation for MGTA-456, an expanded CD34+ cell therapy treatment used in place of single umbilical cord blood (UCB) transplantation. This one-time stem cell treatment is being investigated for the treatment of multiple inherited metabolic disorders. While there are 36 publicly announced RMAT designations, the FDA states that 40 or more have been awarded to date. [Read more…]
SanBio, a company developing regenerative cell medicines for neurological disorders, has been awarded a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for SB623.
SB623 is a cell therapy that the company has developed for the treatment of chronic neurological motor deficits that occur as a result of traumatic brain injury (TBI). In addition to this treatment, SanBio is developing regenerative therapies for stroke, diseases of the eye, Parkinson’s disease (PD), and other neurological conditions. [Read more…]
Three-Year Registrational Data Set to be Presented by Year End 2019
Regulatory Submissions Planned in the U.S. and Europe for 2021
BOSTON and LONDON, July 29, 2019 — Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for OTL-103, Orchard’s ex vivo autologous hematopoietic stem cell (HSC)-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS) developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. [Read more…]
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. [Read more…]
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.