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BridgeBio Pharma and Sentynl Therapeutics Receive Marketing Authorization in the EU for NULIBRY® (fosdenopterin) for the Treatment of MoCD Type A
- The first and only treatment in Europe to treat patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- The first and only treatment in Europe to treat patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants with a median overall survival age of about four years. - European Commission (EC) decision is based on the efficacy and safety data collected to date compared to data from a natural history study. - NULIBRY was BridgeBio’s first FDA-approved therapeutic; Sentynl acquired global rights to NULIBRY in March 2022. PALO ALTO, Calif. and SOLANA BEACH, Calif. and AHMEDABAD, India, Sept. 20, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases owned by Zydus Lifesciences Ltd. (formerly known as Cadila Healthcare Ltd.), today announced the European Commission (EC) has granted marketing authorization for NULIBRY® (fosdenopterin) for Injection as the first therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A. MoCD Type A is an ultra-rare and progressive condition known to impact less than 150 patients globally with a median survival of four years. NULIBRY is a first-in-class cPMP substrate replacement therapy that was approved by the U.S. Food and Drug Administration (FDA) in 2021 to reduce the risk of mortality in patients with MoCD Type A. Following this decision by the EC, NULIBRY is the first and only approved therapy in the European Union (EU) for MoCD Type A. In March 2022, Sentynl acquired the global rights to NULIBRY and is responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing, and commercializing fosdenopterin globally. Sentynl and BridgeBio share development responsibilities through the approval of the marketing authorization application under accelerated assessment with the European Medicines Agency (EMA) and through approval of NULIBRY’s regulatory submission with the Israeli Ministry of Health. \"The European Commission's approval of NULIBRY is an exciting step in delivering this therapy to all children suffering with MoCD Type A worldwide, and it bolsters o...