MetrioPharm’s lead compound, MP1032, has received orphan drug designation (ODD) from the European Medicines Agency (EMA) for treating Duchenne muscular dystrophy (DMD) in children.
This follows the ODD granted by the US Food and Drug Administration (FDA) and highlights MP1032’s potential as a first-in-class therapy that may reduce the need for high-dose corticosteroids, which can lead to serious side effects such as growth stunting, osteoporosis and metabolic issues.
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The EMA’s designation offers significant incentives, such as protocol assistance, fee waivers and market exclusivity of up to 12 years in the European Union (EU) — extended from the standard 10 years due to the paediatric focus on DMD.
The validation is supported by strong preclinical data demonstrating MP1032’s capacity to sustain the function of muscle and minimise inflammation while maintaining normal cellular processes.
MetrioPharm CEO Thomas Christély stated: “This dual ODD from the FDA and EMA, as well as the already received rare paediatric disease designation from the FDA, are game changers for MetrioPharm and the DMD community.
“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.”
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By GlobalDataMP1032 targets the overactive responses of immune cells by modifying a key metabolic process within them.
Upon activation, the mitochondria increase their energy metabolism, resulting in the production of excessive reactive oxygen species.
This anti-inflammatory drug offers therapeutic effects comparable to corticosteroids but without the serious side effects.
