BridgeBio Pharma, Inc , 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. 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 our belief at BridgeBio that no disease is too rare to address. We are grateful that the European Commission sees the value of this treatment, and to the patients, caregivers, physicians, scientists, and advocates whose efforts made this possible," said BridgeBio founder and CEO Neil Kumar, Ph.D.
The EC authorization follows the positive opinion granted by European Committee for Medicinal Products for Human Use (CHMP) in July 2022, which was supported by data from three clinical trials that demonstrated the efficacy of NULIBRY for the treatment of patients with MoCD Type A compared to data from a natural history study. These studies showed that in the genotype-matched analysis, patients treated with NULIBRY had 7.1 times lower risk of death than their historical control counterparts from the natural history study, with 86% of NULIBRY-treated patients surviving at three years compared to 52% of the genotype-matched control group.
“The approval of NULIBRY by the European Commission is a promising development for children with MoCD Type A. Zydus Group is committed to making a meaningful difference in the lives of people suffering from rare and orphan diseases. This approval brings us closer to realizing our purpose of empowering people with the freedom to live healthier and more fulfilled lives,” said Dr. Sharvil Patel, Managing Director of Zydus Lifesciences, the parent company of Sentynl Therapeutics.
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 our belief at BridgeBio that no disease is too rare to address. We are grateful that the European Commission sees the value of this treatment, and to the patients, caregivers, physicians, scientists, and advocates whose efforts made this possible," said BridgeBio founder and CEO Neil Kumar, Ph.D. The EC authorization follows the positive opinion granted by European Committee for Medicinal Products for Human Use (CHMP) in July 2022, which was supported by data from three clinical trials that demonstrated the efficacy of NULIBRY for the treatment of patients with MoCD Type A compared to data from a natural history study. These studies showed that in the genotype-matched analysis, patients treated with NULIBRY had 7.1 times lower risk of death than their historical control counterparts from the natural history study, with 86% of NULIBRY-treated patients surviving at three years compared to 52% of the genotype-matched control group. “The approval of NULIBRY by the European Commission is a promising development for children with MoCD Type A. Zydus Group is committed to making a meaningful difference in the lives of people suffering from rare and orphan diseases. This approval brings us closer to realizing our purpose of empowering people with the freedom to live healthier and more fulfilled lives,” said Dr. Sharvil Patel, Managing Director of Zydus Lifesciences, the parent company of Sentynl Therapeutics. “This is a major milestone for those patients living with MoCD Type A in Europe,” said Matt Heck, CEO of Sentynl. “Marketing authorization is an important step in providing access to NULIBRY and creating awareness of MoCD Type A as many patients are often missed.”
The EC's centralized marketing authorization is valid in all EU member states as well as Iceland, Liechtenstein, and Norway. A regulatory filing is expected in the coming months to the UK's Medicine and Healthcare products Regulatory Agency (MHRA) as part of the European Commission Decision Reliance Procedure. Sentynl expects to make NULIBRY available following successful completion of country-by-country health authority discussions. Until such time, NULIBRY will be made available to qualified patients through an Early Access Program.
