Switzerland-based EspeRare Foundation has received an orphan drug designation (ODD) from the European Medicines Agency (EMA) for its investigational compound rimeporide to treat patients with Duchenne muscular dystrophy (DMD).
DMD is a rare genetic paediatric disease, which causes muscle weakness and muscle loss. It affects approximately one in 3,500 male babies worldwide.
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Rimeporide is a selective sodium/proton exchanger type-1 inhibitor, which was originally developed by Merck Serono in the intended indication of congestive heart failure.
EspeRare R&D director Florence Porte-Thomé said: "Rimeporide’s potential to address skeletal muscle inflammation, fibrosis and cardiomyopathy in a broad population of patients, regardless of their mutational status, could make it an ideal complement to treatments designed to augment or replace dystrophin.
"Obtaining this ODD is an important milestone for rimeporide and supports its clinical development."
EspeRare received rights for rimeporide in 2013, and it successfully carried out two non-clinical studies at the Children Medical Centre in Washington, US, and the University of Geneva, Switzerland.
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By GlobalDataRimeporide demonstrated a clinically acceptable safety profile in adults, in clinical studies to date, according to EspeRare.
EspeRare plans to start the first clinical trial in boys with DMD during the second half of this year to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of this drug candidate in these patients.
Paris Hospital Cochin cardiologist professor Denis Duboc said: "The nonclinical package indicates rimeporide’s potential to transform Duchenne muscular dystrophy from a life-threatening to a chronic disease."
Image: The FDA has granted orphan drug designation to EspeRare’s investigational compound rimeporide. Photo: courtesy of EspeRare Foundation.
