Tuesday, August 08, 2023
SYSNAV Healthcare has achieved a significant milestone in the healthcare field with the European Medicines Agency (EMA) endorsing the use of Stride Velocity 95th centile (SV95C) as a primary endpoint in superiority studies for ambulant Duchenne Muscular Dystrophy (DMD) patients aged four and above. This regulatory approval offers an alternative to the 6 Minute Walking Test, subject to consistent findings in established efficacy endpoints within secondary endpoints. This recognition makes SV95C the first digital endpoint to receive regulatory endorsement for its application as a primary endpoint in pivotal studies.
This accomplishment is the result of a fourteen-year journey that involved collaboration among various stakeholders within the DMD community, including technology developers, academia, the pharmaceutical industry, caregivers, and most importantly, DMD patients and their families.
Duchenne Muscular Dystrophy is a rare and progressive neuromuscular disorder that leads to muscle weakness and wheelchair dependency for most patients by the age of 13. While promising therapeutic advancements are on the horizon, evaluating the impact of interventions can be challenging due to the variable progression of the disease. Objective and rigorous treatment efficacy assessment is crucial, and SV95C, as a real-world digital endpoint, fills this critical gap, potentially expediting studies, reducing participant requirements, and streamlining the approval process.
Damien Eggenspieler, SYSNAV Healthcare's Program Director, highlighted the transformative potential of digital endpoints in clinical development. SV95C, by providing a more precise measurement of functional ability in DMD patients and enabling a more objective assessment of treatment benefits, has the capacity to shorten the duration of clinical trials and potentially reduce the number of participants needed. The collaborative efforts of SYSNAV and industry partner Roche played a vital role in securing the regulatory qualification of SV95C. Their partnership aims to extend the use of approved endpoints to several priority diseases within their portfolio, while actively involving patient communities in the process.