MetrioPharm announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD).
This milestone follows the U.S. FDA’s ODD, underscoring MP1032’s potential as a first-in-class therapy that could minimize reliance on high-dose corticosteroids, which cause severe side effects like osteoporosis, growth stunting, and metabolic issues.
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Key Findings of MetrioPharm MP1032
- European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)
- Designation is paving the way for safer long-term therapy
- MP1032 has demonstrated ability to preserve muscle function and reduce inflammation without disrupting normal cellular processes
The EMA’s ODD provides key incentives, including protocol assistance from the EMA, fee waivers, and up to 12 years of market exclusivity in the EU (extended from 10 years due to DMD’s pediatric focus) upon approval. This regulatory validation builds on compelling preclinical data showing MP1032’s ability to preserve muscle function and reduce inflammation without disrupting normal cellular processes.
“This dual ODD from the FDA and EMA as well as the already received Rare Pediatric Disease Designation from the FDA are game changers for MetrioPharm and the DMD community,” said CEO Thomas Christély. “MP1032’s unique mechanism — modulating mitochondrial ROS in overactive immune cells — could transform long-term DMD management by slashing corticosteroid doses and side effects. We’re accelerating MP1032 towards Phase II trials, collaborating with patient groups, and exploring partnerships to bring this to children who need safer options. Beyond DMD, our platform’s host-directed approach has shown promise in autoimmune diseases and even pandemics, as evidenced by our HERA-funded COVID-19 Phase IIa study.”
What is MetrioPharm MP1032?
MetrioPharm’s lead candidate MP1032 is a unique small molecule targeting overshooting reactions of activated immune cells by addressing a central, natural metabolic process occurring in activated immune cells. The mitochondria of these cells react to the activation by boosting their energy metabolism, which in turn leads to the generation of excessive reactive oxygen species (ROS). MP1032 is a first-in-class anti-inflammatory drug that reduces excessive ROS without interfering with the physiological ROS levels of other cells, that are essential for normal cell function, resulting in an excellent safety profile. As a monotherapy, MP1032 produces therapeutic effects similar to corticosteroids (cortisone-based therapeutics), but without serious side effects. Unlike traditional anti-inflammatories, it mimics corticosteroid benefits as a monotherapy but avoids their toxicities, as demonstrated in preclinical models of DMD, multiple sclerosis, arthritis, and more.
