BridgeBio's BBP-812 Canavan Disease Gene Therapy Receives FDA RMAT Designation

(RTTNews) - BridgeBio Pharma Inc. (BBIO) announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy or RMAT designation to BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease.

The 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 noted that it 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.

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