BridgeBio Pharma and Sentynl Therapeutics Receive Marketing Authorization in the EU for NULIBRY®
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)
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)
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.
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.
“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.
Sentynl expects to make NULIBRY available following successful completion of country-by-country health authority discussions.
I n July 2022, BridgeBio received New Drug Application (NDA) Approval of NULIBRY as a treatment for MoCD Type A from the Israeli Ministry of Health.
It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group.
1,2 The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.
Based on these estimates, MoCD Type A is likely to be underdiagnosed.
This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.
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