Drug that could be the first and only in Europe to treat rare brain condition recommended for marketing authorization

brain stroke

The Committee for Medical Products for Human Use (CHMP) has recommended the European Commission (EC) make a marketing authorization under exceptional circumstances for a drug to treat a rare condition characterized by brain dysfunction.

The drug is called NULIBRY (fosdenopterin) for injection therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.

Molybdenum cofactor deficiency (MoCD) type A is a rare but devastating metabolic disease and first appears in the newborn period. It progresses rapidly and causes severe developmental delay within the first four years of life. It occurs through a gene abnormality.

Toxic sulfite

The changed gene causes a toxic sulfite to collect in the brain. The sulfite leads to brain dysfunction. An early diagnosis is critical to any chance of improving outcomes.  

BridgeBio Pharma, Inc.,  a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc., a U.S.-based biopharmaceutical company that produces therapies for people living with rare diseases made the announcement today (July 25).

NULIBRY is a 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.

Global rights

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 EMA and through approval of NULIBRY’s regulatory submission with the Israeli Ministry of Health.

Neil Kumar, CEO at BridgeBio, said: “Our work on NULIBRY and MoCD Type A epitomizes BridgeBio’s belief that no disease is too rare to address. With this positive CHMP opinion, we are closer to delivering a treatment option to all children across the globe who suffer with MoCD Type A.”

Reduced risk of death

The positive CHMP opinion is supported by data from three clinical trials that demonstrated efficacy of NULIBRY for the treatment of patients with MoCD Type A compared to data from a natural history study. These studies showed that NULIBRY reduced the risk of death by 86% and increased the probability of survival to 86% at three years compared to 52% in the untreated, genotype-matched, historical control group in the natural history study.

Matt Heck, CEO at Sentynl said: “We are thrilled by the CHMP’s recommendation in favor of NULIBRY and hope that patients living with MoCD Type A in Europe and around the world can access this therapy. The CHMP positive opinion marks important progress not only for the program but also for the MoCD Type A patients outside of the U.S. who are seeking ways to treat their life-threatening and progressive disease.”

Based on the CHMP recommendation, a decision by the EC, which authorizes marketing applications in the EU, is expected on the NULIBRY application later this year. The recommendation for marketing authorization under exceptional circumstances is granted to medicines where the applicant is unable to provide comprehensive data under normal conditions of use because the disease being treated is so rare.

In April 2022, BridgeBio received New Drug Application (NDA) Approval in Principle from the Israeli Ministry of Health and the application is currently undergoing the final review processes.

Progressive brain damage

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). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.

NULIBRY®(Fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites.

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.

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