BridgeBio Gets FDA RMAT Designation for BBP-812 Canavan Disease Gene Therapy

BridgeBio Pharma, Inc., a biopharmaceutical company dedicated to addressing genetic diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BBP-812. BBP-812 is an investigational gene therapy utilizing an adeno-associated virus serotype 9 (AAV9) vector, aimed at treating Canavan disease. This designation is based on encouraging preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which demonstrated functional improvements in all dosed patients, suggesting that BBP-812 could meet the significant unmet needs of individuals with Canavan disease.

RMAT designation is an expedited FDA program designed for regenerative medicine therapies intended to treat, modify, reverse, or cure serious conditions. It offers the benefits of the Fast Track and Breakthrough Therapy Designation programs, enabling more rapid and frequent interactions with the FDA to facilitate early alignment on critical program aspects. The RMAT designation was granted following the FDA’s review of 12 months of safety and efficacy data from the first eight patients with Canavan disease treated in the CANaspire Phase 1/2 trial.

Eric David, M.D., J.D., CEO of BridgeBio Gene Therapy, expressed gratitude for the RMAT designation, emphasizing the company’s commitment to working closely with the FDA and the Canavan community to bring the therapy to affected families as swiftly as possible. David extended his appreciation to the children and families participating in the CANaspire study, as well as the study investigators.

The CANaspire trial results to date have shown that all patients receiving BBP-812 with at least one follow-up assessment have exhibited functional improvements in critical areas such as head control, sitting upright, reaching for and grasping objects, and visual tracking. Additionally, these patients have demonstrated reductions in N-acetylaspartate (NAA) levels, both in urine and the central nervous system, to levels associated with mild disease. BBP-812 has been well-tolerated, with a safety profile consistent with other AAV9 gene therapy programs.

Canavan disease is a rare and rapidly progressing neurodegenerative disorder that hinders children from achieving basic developmental milestones, including crawling, walking, speaking, and even holding their heads up. It is a terminal diagnosis with no approved treatments currently available. Kathleen Flynn, CEO of the National Tay-Sachs & Allied Diseases Association, highlighted the significance of the RMAT designation and the promising results from the clinical trial, offering hope to children and families affected by Canavan disease globally.

In addition to the RMAT designation, BBP-812 has received Orphan Drug, Rare Pediatric Disease (RPDD), and Fast Track Designations from the FDA, as well as Orphan Drug Designation from the European Medicines Agency. If approved, the RPDD could qualify BridgeBio for a Priority Review Voucher.

The CANaspire trial is an open-label Phase 1/2 study designed to assess the safety, tolerability, and pharmacodynamic activity of BBP-812 in pediatric patients with Canavan disease. Each eligible patient receives a single intravenous infusion of BBP-812, with primary outcomes including safety and changes in urine and central nervous system NAA levels, as well as assessments of motor function and development.

Canavan disease affects approximately 1,000 children in the United States and the European Union. The disease is caused by an inherited mutation of the ASPA gene, which encodes the enzyme aspartoacylase. The deficiency of aspartoacylase activity leads to the accumulation of NAA, resulting in toxicity to myelin, which insulates neuronal axons. Without proper myelination, neurons cannot effectively send and receive messages. Current care for Canavan disease is limited to supportive therapy.

BridgeBio Pharma, Inc. is a commercial-stage biopharmaceutical company founded in 2015 to develop transformative medicines for genetic diseases. The company’s pipeline includes programs ranging from early-stage science to advanced clinical trials, driven by a team committed to advancing genetic medicine to benefit patients as rapidly as possible.

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