DALLAS—Vamorolone therapy may impair growth less than other therapies do in patients with Duchenne muscular dystrophy (DMD), according to a study presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.
“Children with DMD are on average shorter than the general population by the age of 5 years, and daily dosing with prednisone or deflazacort leads to further growth stunting,” wrote Dr. Stanley Iyadurai of Catalyst Pharmaceuticals and coauthors of the study.
They described vamorolone, which is marketed by Catalyst, as “a novel drug with a chemical structure distinct from classic corticosteroids.” It was approved in the U.S. in 2023 for treating DMD in patients who are 2 years old or older.
For the study, researchers analyzed results from several trials of varying lengths to compare growth velocities in patients with DMD treated with vamorolone, prednisone, deflazacort or placebo.
A 24-week analysis of the VISION-DMD trial found similar growth velocities for patients who received prednisone and vamorolone (2 mg/kg or 6 mg/kg daily) compared with a placebo, according to the study. However, patients who received vamorolone at 6 mg/kg daily had significantly higher growth velocity than patients receiving prednisone.
Read more about signs and symptoms of DMD
A 48-week analysis of the VISION-DMD and FOR-DMD randomized trials revealed significantly higher growth velocities for patients receiving 2 mg/kg or 6 mg/kg of vamorolone daily, compared with patients receiving prednisone or deflazacort.
Finally, in a 2.5-year analysis of the open-label VBP15-LTE trial and the FOR-DMD study, patients who received 2-6 mg/kg of vamorolone daily had significantly higher growth velocities compared with patients treated with prednisone or deflazacort.
“The results of this post hoc analysis suggest that vamorolone does not appear to impair linear growth as do prednisone or deflazacort,” researchers concluded.
