Pharma Focus America

MetrioPharm Granted Orphan Drug Designation for Duchenne Muscular Dystrophy Treatment

Tuesday, May 30, 2023

MetrioPharm AG, a pharmaceutical company specializing in the development of drugs for inflammatory and infectious diseases, has recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to their drug candidate MP1032 for the treatment of Duchenne muscular dystrophy (DMD).

DMD is the most prevalent form of muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration. The symptoms typically manifest in early childhood, usually between the ages of two and three. The disease primarily affects boys, leading to severe muscle loss and heart failure, while girls can experience milder symptoms. DMD significantly reduces life expectancy. Current standard therapies involve long-term administration of high-dose corticosteroids, which can only slow down disease progression and have significant side effects that impact patients' quality of life.

MetrioPharm aims to address the limitations of current treatments for DMD with their drug MP1032. Their Chief Scientific Officer and co-founder, Dr. Wolfgang Brysch, highlighted that while DMD cannot currently be cured, it can be treated. The goal of MP1032 is to improve the tolerability of treatment while further slowing down disease progression, ultimately enhancing both safety and efficacy for long-term DMD patients.

Orphan Drug Designation is granted by the FDA to drug candidates that show promise in treating rare medical diseases affecting fewer than 200,000 people in the U.S. This designation provides various benefits to drug developers, including development assistance, FDA fee exemptions, and seven years of marketing exclusivity after approval. For MetrioPharm, obtaining Orphan Drug Designation for MP1032 in DMD is a significant achievement that brings them closer to obtaining accelerated market approval for the drug in Duchenne patients.

MetrioPharm plans to initiate a Phase II clinical trial for MP1032 in DMD in 2024. The company has conducted preclinical studies in collaboration with the patient organization Duchenne UK. These studies involved testing MP1032 in an mdx model for DMD and comparing its effects to those of corticosteroids. The results demonstrated that MP1032 was able to enhance muscle strength similarly to the corticosteroid Prednisolone® but without its associated serious side effects. Another preclinical study conducted by Eurofins revealed that the combination of MP1032 with a significantly reduced dose of Prednisolone® exhibited an efficacy increase of more than two and a half times the normal dose of Prednisolone®. This synergistic effect suggests that the combined use of MP1032 and Prednisolone® may be more effective than either substance alone.

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