Ahmedabad, Sep 20 Zydus Lifesciences Limited-owned US-based Sentynl Therapeutics, Inc and BridgeBio Pharma, Inc, jointly announced that 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.
Sentynl Therapeutics is the manufacturer of innovative therapies for rare diseases, while BridgeBio Pharma, Inc is a biopharmaceutical company focused on genetic diseases and cancers.
Rare and progressive disease
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. The European Commission (EC) decision is based on the efficacy and safety data collected till date compared to data from a natural history study, a company statement said.
NULIBRY is a first-in-class cyclic pyranopterin monophosphate (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 its commercialization in the US and also developing, manufacturing, and commercializing fosdenopterin globally, a statement said.
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.
“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 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.
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.
In July 2022, BridgeBio had received New Drug Application (NDA) approval of NULIBRY as a treatment for MoCD Type A from the Israeli Ministry of Health.
MoCD Type A disease
MoCD Type A is an ultra-rare disease. The incidence and prevalence of MoCD Type A in the European Union are not known, but the estimated prevalence is 0.005 per 10,000. Based on these estimates, MoCD Type A is likely to be underdiagnosed.
The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI).
NULIBRY®(fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin.
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.