Drug could be first and only in Europe to treat rare brain disease

EThe Committee for Medicinal Products for Human Use (CHMP) has recommended that the European Commission (EC) grant marketing authorization in exceptional circumstances for a medicine intended to treat a rare disease 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 neonatal period. It progresses rapidly and causes severe developmental delay during the first four years of life. It occurs by a genetic abnormality.

toxic sulfite

The modified gene causes the accumulation of a toxic sulfite in the brain. Sulfite causes brain dysfunction. Early diagnosis is essential for 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 US-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 United States 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 therapeutic approved in the EU for MoCD type A.

Global rights

In March 2022, Sentynl acquired the worldwide rights to NULIBRY and is responsible for the ongoing development and commercialization of NULIBRY in the United States and the development, manufacturing and commercialization of fosdenopterin worldwide. Sentynl and BridgeBio share development responsibilities through approval of the accelerated review marketing authorization application with the EMA and through approval of NULIBRY’s regulatory submission with the Israeli Ministry of Health.

Neil Kumar, CEO of BridgeBio, said, “Our work on NULIBRY and MoCD Type A embodies BridgeBio’s belief that no disease is too rare to treat. With this positive opinion from the CHMP, we are one step closer to providing a treatment option for all children around the world who suffer from MoCD type A.”

Reduced risk of death

The positive CHMP opinion is supported by data from three clinical trials which 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 NULIBRY reduced the risk of death by 86% and increased the probability of survival to 86% at three years, compared to 52% in the historical untreated control group, of the same genotype, in the natural history study. .

Matt Heck, CEO of Sentynl, said: “We are delighted with the CHMP recommendation for NULIBRY and hope that patients living with MoCD type A in Europe and globally will be able to access this therapy. The positive CHMP opinion marks significant progress not only for the program, but also for MoCD Type A patients outside the United States who are seeking ways to treat their progressive and life-threatening disease.

Based on the CHMP recommendation, an EC decision, which allows marketing applications in the EU, is expected on the NULIBRY application later this year. The recommendation for marketing authorization in exceptional circumstances is granted to medicinal products for which the applicant is unable to provide complete data under normal conditions of use due to the rarity of the disease being treated.

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

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 as characteristic patterns on magnetic resonance imaging (MRI). This damage results in severe psychomotor impairment and an inability to perform 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 into molybdenum cofactor, which is necessary 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 treatment indicated to reduce the risk of mortality in patients with MoCD type A, and clinical trials have shown that patients treated with NULIBRY or rcPMP had improved overall survival compared to untreated patients of the same genotype. , historical control group.

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