EMA gives nod to Critical Path Institute's trial simulation platform for DMD

The European Medicines Agency sent a letter of support for Critical Path Institute’s efforts to establish a model-based clinical trial simulation platform that could help find new treatments for Duchenne muscular dystrophy (DMD).

The regulatory agency issued the letter to encourage further development and validation of the platform and encourage sponsors to share patient-level data with the Duchenne Regulatory Science Consortium (D-RSC). Critical Path, which is an independent nonprofit, public-private partnership with the FDA to improve and streamline medical product development, co-founded D-RSC with fellow non-profit Parent Project Muscular Dystrophy.

Critical Path’s platform will aid in exploring key design constructs that include sample size, sampling scheme, patient enrollment criteria, dose and study design, as well as accelerating medical product development, the group said in a Jan. 25 release.

“This letter of support is an outstanding milestone and a testament to the D-RSC members’ continued support in providing strong scientific leadership and pioneering data sharing for the benefit of the entire Duchenne community,” Klaus Romero, M.D., Critical Path’s chief science officer, said in the release.

DMD is a fatal genetic disorder that leads to worsening muscle wasting over time and primarily occurs in males diagnosed between ages the ages of two and five years.

In early January, the CDMO Catalent inked a deal with Sarepta Therapeutics to manufacture a gene therapy to treat DMD. The therapy is designed to deliver the microdystrophin-encoding gene into muscle tissue that triggers production of the microdystrophin protein.

The FDA approved Sarepta’s biologics license application in November seeking accelerated approval of the therapy, dubbed delandistrogene moxeparvovec. If given final sign-off by the agency, it would be the first gene therapy available to treat the condition.