Duchenne Muscular Dystrophy research receives EMA support

The EMA has published a letter of support for the Duchenne Regulatory Science Consortium’s (D-RSC) Clinical Trial Simulation Platform (CTSP) for Duchenne Muscular Dystrophy (DMD) treatments.

In the letter, published in 2022, the EMA stated that it has acknowledged the Critical Path Institute’s efforts to establish the CTSP for DMD treatments and has seeks to encourage the further development and validation of the CTSP.

DMD is a rare, muscle-wasting disease that results from the deficiency of a protein called dystrophin. Those affected experience loss of muscle tissue with consequent muscle weakness, loss of ambulation, loss of upper body function and more.

Due to its rarity and inherent complexity, optimal design of DMD clinical trials remains a field-wide challenge. D-RSC’s simulation platform – the first of its kind for DMD – will help advise on key design constructs for the trials including patient enrolment criteria, sample size, and dose.

D-RSC’s Executive Director, Terina Martínez, said that the EMA’s letter is a huge accomplishment and highlights the D-RSC team’s continued efforts to accelerate drug development for DMD.