BridgeBio Pharma Inc. (BBIO) has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BBP-812, an experimental intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for treating Canavan disease.
This RMAT designation is based on early clinical evidence from the CANaspire Phase 1/2 trial, which demonstrated functional improvements in all treated patients, suggesting that BBP-812 has the potential to meet the unmet needs of individuals with Canavan disease.
BridgeBio highlighted that it will utilize the benefits of the RMAT designation, such as earlier and more frequent interactions with the FDA, to establish a pathway for the Accelerated Approval of BBP-812.
If approved, BridgeBio's gene therapy could become the first therapeutic option for children born with Canavan disease, a severe and fatal neurodevelopmental disorder.
