Advancing treatments for rare diseases Together

DMD

04Oct

The last advances of the Rimeporide clinical study have been presented at the 22nd World Muscle Society congress, in St Malo. Numerous scientists and physicians have shown interest for this project.

Enrollment into this phase IB clinical trial should be complemented by the end of the year. Full results should be available during the first quarter of 2018. EspeRare is initiating discussions with  worldwide clinicians in order to design the phase II study which should start during 2018, depending  on funding.

Poster presented at the World Muscle Society congress

 

26Sep

The Food and Drug Administration (FDA), the US Department of Health and Human Services, has granted an Orphan Drug Designation (ODD) for rimeporide for the treatment of Duchenne muscular dystrophy (DMD). See here for more details.

ODD is designed to promote the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. In particular, this ODD grants Rimeporide a 7-year data exclusivity in the US.

Rimeporide already received ODD in DMD from the European Medicines Agency in 2015. Further to this designation in Europe, EspeRare has been able to conduct a phase Ib clinical trial of Rimeporide on young DMD patients. This clinical trial will be completed by the end of 2017.