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BridgeBio Pharma and Sentynl Therapeutics Receive Positive CHMP Opinion for NULIBRY® (fosdenopterin) for the Treatment of MoCD Type A
- CHMP recommendation for approval of NULIBRY in the European Union (EU) for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A is
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- CHMP recommendation for approval of NULIBRY in the European Union (EU) for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A is based on the efficacy and safety data collected to date compared to data from a natural history study \n- Under an accelerated assessment pathway, a decision by the European Commission (EC), which authorizes marketing approval in the European Union (EU), is expected on the NULIBRY application later this year\n- If approved by the EC, NULIBRY would be the first and only approved therapy in the EU to treat patients with 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\n- NULIBRY was BridgeBio's first FDA-approved therapeutic; Sentynl acquired global rights to NULIBRY in March 2022 \nPALO ALTO, Calif. and SOLANA BEACH, Calif., July 25, 2022 /PRNewswire/ -- 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 that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended that the European Commission authorize marketing under exceptional circumstances 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.\nBridgeBio Pharma and Sentynl Therapeutics Receive Positive CHMP Opinion for NULIBRY® (fosdenopterin)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. If approved by the European Commission, NULIBRY would be the first and only approved therapy in the EU for MoCD Type A.\nIn March 2022, Sentynl acquired the global rights to NULIBRY and is responsible for the ongoing development and commercialization of NULIBRY i...