FDA Grants Special Therapy Designation to BridgeBio for Achondroplasia Treatment

September 17, 2024 – BridgeBio Pharma, Inc.

BridgeBio Pharma announced that the U.S. Food and Drug Administration (FDA) has granted a special therapy designation to its oral treatment under development for children with achondroplasia, the most common form of dwarfism, which impacts skeletal development and overall health.

The designation was awarded based on data from the Phase 2 PROPEL 2 study. In the study, children in Cohort 5 receiving 0.25 mg/kg/day of the treatment experienced a significant increase in annualized height velocity (AHV), with a mean increase of +2.51 cm/year at 12 months, sustained at 18 months. Additionally, improvements in body proportionality were noted at 18 months.

Advocacy groups have welcomed the development. Munira Shamim, founder of Growing Stronger, an advocacy organization for those affected by achondroplasia, highlighted that this designation could provide families with more treatment options in managing the condition.

With this designation, BridgeBio aims to expedite the development and regulatory review of the treatment. If approved, it would be the first oral therapy available for achondroplasia. The treatment has also received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA.

The Phase 3 PROPEL 3 study is currently underway, with completion of enrollment expected by the end of 2024. More information is available on clinicaltrials.gov (NCT06164951).

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Related Services