Sarepta Therapeutics has announced its plans to request an expansion of its Duchenne muscular dystrophy (DMD) treatment to patients of all age groups. The decision comes after the company's study showed promising results.
The study evaluated the efficacy of Elevidys, Sarepta's treatment for DMD, by measuring motor function using the North Star Ambulatory Assessment. Although the study did not meet its primary endpoint, patients treated with Elevidys demonstrated an increase in motor function compared to those who received a placebo after 52 weeks.
The findings from the study support Sarepta's belief that Elevidys can alter the trajectory of individuals living with Duchenne. The Phase 3 clinical trial focused on patients between the ages of four and seven.
In June, Elevidys received accelerated FDA approval for patients aged four to five, marking an important milestone in DMD treatment.
Based on the positive results of the study, Sarepta intends to swiftly request an update to expand the labeled indication to treat all patients. Chief Executive Doug Ingram confirmed that the company has received support from the leadership at the Food and Drug Administration, indicating their openness to such an expansion pending a thorough review of the data.
It is evident that Sarepta Therapeutics is dedicated to advancing the treatment options available for Duchenne muscular dystrophy, bringing hope to patients of all ages.
