EspeRare Receives European Orphan Drug Designation for Rimeporide in Duchenne Muscular Dystrophy

EspeRare, a company focused on uncovering the potential of existing molecules to address severe therapeutic unmet needs in rare diseases, recently received European Medicines Agency (EMA) Orphan Drug Designation for their Rimeporide compound for the treatment of Duchenne Muscular Dystrophy (DMD).

DMD is a rare, inherited, life-threatening disorder that involves muscle weakness hat quickly progresses. DMD is the most common and severe form of pediatric muscular dystrophies.

EspeRare’s Rimeporide is a selective inhibitor of sodium/proton exchanger type-1 that was initially developed by Merck Serono for the treatment of congestive heart failure. Results from clinical trials conducted in adults to assess the efficacy of the compound showed that it has an acceptable safety profile. In 2013, the rights of rimeporide were transferred to EspeRare. Since then, the company has conducted two non-clinical trials at the University of Geneva in Switzerland and also at the Children Medical Centre in Washington. Last year, at the ICNMD XIII Congress on Neuromuscular Diseases in Nice, France, the company presented the results of these trials.

“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” commented Florence Porte-Thomé, EspeRare’s R&D Director, in a recent press release.

EMA’s decision to grant Orphan Medicinal Products to rimeporide represents the first time that an inhibitor of sodium/proton exchange receives orphan status for DMD in Europe.

Based on the results of the safety and efficacy reported in the non-clinical studies, the company is going to launch during the second semester of 2015 the first clinical trial in boys with DMD to examine the pharmacodynamics and the safety tolerability pharmacokinetics of rimeporide in this patient population.

“The nonclinical package indicates rimeporide’s potential to transform Duchenne muscular dystrophy from a life-threatening to a chronic disease” explained Professor Denis Duboc Cardiologist at the Hospital Cochin in Paris.

The grant of Orphan Drug Designation for Rimeporide demonstrates the compound dormant therapeutic assets as a potential treatment for patients suffering from orphan diseases. This achievement motivates EspeRare to further build on its model by expanding its collaborations with biopharmaceutical companies and operating towards building a vigorous portfolio of programs for patients disturbed by these conditions.