- FDA grants orphan drug designation for MetrioPharm’s lead compound MP1032 for the treatment of Duchenne muscular dystrophy (DMD)
- DMD is a designated orphan disease in both Europe and the U.S.
- MetrioPharm’s development objectives: better tolerability of existing therapies and improved efficacy in long-term treatment of DMD patients
Zurich, May 30, 2023 – MetrioPharm AG, a pharmaceutical company developing drugs for inflammatory and infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for MP1032 for the treatment of Duchenne muscular dystrophy.
DMD is the most common form of muscular dystrophy. It is a genetic disorder characterized by progressive muscle degeneration; symptom onset is in early childhood, usually between the ages of two and three. The disease primarily affects boys causing severe muscle loss and heart failure, while girls can show milder symptoms as well. DMD shortens life expectancy significantly. Existing standard therapies consist of treatment with high dose corticosteroids for decades that lead to serious side-effects and can only slow disease progression.
“Currently, DMD cannot be cured, but it can be treated,” said MetrioPharm CSO and co-founder Dr. Wolfgang Brysch. “These treatments have serious side effects that heavily impact patients’ quality of life. With MP1032 we aim to improve the tolerability of treatment while also further slowing down disease progression. Our goal is to improve both safety and efficacy in the long-term treatment of DMD patients.”
“The orphan drug designation is granted by the FDA for drug candidates that the FDA considers a promising new treatment. In a designated orphan development, timelines are shorter, and costs are lower compared to indications with larger patient populations,” said MetrioPharm CEO Thomas Christély. “The Orphan Drug Designation for MP1032 in DMD by the FDA is a very important achievement; it takes us one crucial step closer…