EMA Approves First Digital Outcome Measure as Primary Endpoint in Pivotal Trials

Rare Daily Staff

The European Medicines Agency said that it will accept a digital endpoint for ambulant Duchenne muscular dystrophy patients who are 4 years old and older as a primary endpoint in superiority.

The EMA said Stride Velocity 95th centile (SV95C) qualifies as primary endpoint in superiority studies as an alternative to the 6 Minute Walking Test provided the outcome measure is supported by consistent findings in established efficacy endpoints included in secondary endpoints. SV95C is the first digital endpoint that has been qualified by a regulatory agency for use as a primary endpoint in pivotal studies.

SV95C is a digital outcome measure of peak ambulatory performance of patients captured during normal daily living. It provides an accurate and clinically meaningful insight into real-world functional ability of patients and measures the progression of their disease or efficacy of therapies.

As an objective and real-world endpoint, SV95C addresses many of the limitations of traditional endpoints currently used in clinical trials in DMD.

This achievement is the result of a fourteen-year collaboration between many stakeholders across the DMD community, including technology developers, academia, the pharmaceutical industry, caregivers, and most importantly patients and families living with DMD.

“It is critical to measure treatment efficacy objectively and rigorously,” said Pat Furlong, president of Parent Project Muscular Dystrophy. “SV95C is this piece of the puzzle, a real-world digital endpoint that has the potential to decrease the duration of studies, lower enrollment requirements, and advance the approval process.”

DMD is a rare and progressive neuromuscular disease-causing muscle weakness that renders the majority of patients wheelchair dependent by the age of 13. While there is significant promise in new therapy development for Duchenne, measuring the impact of an intervention can be difficult due to the heterogeneity of disease progression.

“We believe in the transformative impact of digital endpoints in clinical development—enabling better measurement of the functional ability of DMD patients and to assess potential treatment benefits more objectively and reliably,” said Damien Eggenspieler, SYSNAV Healthcare program director. “The digital endpoint SV95C has the potential to shorten clinical trial duration or decrease the number of patients,”

SV95C can be captured to a regulatory standard by the ActiMyo and Syde wearable devices developed by SYSNAV Healthcare’s proprietary wearable technology and analytical methodology. SYSNAV recently signed a partnership with Roche to expand regulatory approved endpoints beyond DMD to several priority diseases from their portfolio, in partnership with the patient communities.

Photo: Pat Furlong, president of Parent Project Muscular Dystrophy