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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307
2nd Investigational Gene Therapy in Clinic, Potential 3rd Therapy in PKU Franchise, 15+ Year Commitment to PKU Community SAN RAFAEL, Calif., Oct. 2, 2020
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"2nd Investigational Gene Therapy in Clinic, Potential 3rd Therapy in PKU Franchise, 15+ Year Commitment to PKU Community\n\n\nSAN RAFAEL, Calif., Oct. 2, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BMN 307, an investigational gene therapy for the treatment of individuals with PKU.\n\n \n \n \n \n \n \n\n \nFast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation. \n\"Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy,\" said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. \"We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU.\" \nPKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, in many countries, infants are screened at ...