EspeRare is delighted to announce that the United States Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Rimeporide, an investigational treatment for cardiomyopathy in children with Duchenne Muscular Dystrophy (DMD). Most of the drugs in development for DMD, approved or used off label, essentially address the skeletal muscles in DMD patients and are aiming to prolong ambulation. Despite being the primary cause of premature death, there are unfortunately no approved dystrophin specific targeted cardiac therapies.
Rimeporide is a first in class, potent and selective Sodium-Hydrogen Exchanger type 1 (NHE-1) inhibitor. By inhibiting NHE-1, it is expected that Rimeporide modulates the damaging ion imbalances that are associated with the lack of dystrophin in DMD patients. Rimeporide was originally developed as a treatment for congestive heart failure (CHF). Rimeporide has recently been tested in a Phase Ib clinical trial in DMD boys (Previtali et al, 2020).
Rimeporide is intended to be administered as a daily oral chronic treatment, in all patients with DMD, regardless of their mutation, and as soon as early signs of myocardial fibrosis are detected. Pending funding, it is planned to launch a worldwide phase II/III clinical study.
This designation represents an important milestone for Rimeporide and EspeRare as we are now eligible to receive a priority review voucher at the time of marketing authorisation approval of Rimeporide by the US FDA.
