BridgeBio Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for BBP-812 Canavan Disease Gene Therapy Program

- Receipt of RMAT Designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which showed functional improvements in all dosed patients indicating that BBP-812 has potential to address the unmet needs of individuals with Canavan disease

- BridgeBio will leverage the benefits of RMAT designation, including early and more frequent interactions with the FDA, to establish an Accelerated Approval pathway for BBP-812

- If approved, BridgeBio’s gene therapy for Canavan disease could be the first therapeutic option for children born with this devastating and fatal neurodevelopmental disorder

PALO ALTO, Calif., Sept. 10, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease. RMAT designation was granted following the FDA’s review of clinical data from the CANaspire Phase 1/2 clinical trial investigating BBP-812 as a potential therapy to address the unmet medical needs of individuals with Canavan disease.

RMAT is an expedited FDA program available to sponsors of regenerative medicine therapies intended to treat, modify, reverse, or cure serious conditions. Benefits of the RMAT designation include all the advantages of the Fast Track and Breakthrough Therapy Designation programs, including faster and more frequent interactions with the FDA to achieve early alignment on critical aspects of the program. FDA granted RMAT designation based on its review of 12 months of safety and efficacy data from the first eight patients with Canavan disease dosed with BBP-812 in the CANaspire Phase 1/2 clinical trial.

“We are honored to be granted RMAT designation for BBP-812 and are eager to work closely with the FDA and the Canavan community with the goal of bringing our therapy to families living with Canavan disease as fast as possible,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. “We are beyond grateful to the children and their families who are participating in CANaspire, as well as to the study investigators. RMAT will allow us to work more closely with FDA to ensure we are responding to the urgency that families feel.”