Santhera Asks EMA to Approve Vamorolone for DMD
The application is supported by results from a 48-week trial, 3 open-label studies
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Santhera Pharmaceuticals has asked the European Medicines Agency (EMA) to approve vamorolone to treat Duchenne muscular dystrophy (DMD).
The agency’s assessments of new approval requests — in the form of marketing authorization applications — take up to 210 days, but can be longer if the Committee for Medicinal Products for Human Use (an arm of the EMA) has questions or outstanding issues to be addressed.
“Our EU [Europe Union] submission marks an important step towards making vamorolone available to DMD patients in the respective European markets and we look forward to working closely with the EMA during the review process,” Shabir Hasham, MD, Santhera’s chief medical officer, said in a press release. “Duchenne is a debilitating disease with patients in need for effective and well-tolerated therapies.”
DMD is caused by mutations in the gene that provides instructions for producing dystrophin, a protein that helps protect muscle cells from damage during muscle contraction. The condition is usually managed with corticosteroids, anti-inflammatory medications, such as prednisone. Corticosteroids often have a number of side effects, however.
Vamorolone, given orally as a flavored liquid, is a so-called dissociative steroid that maintains corticosteroids’ anti-inflammatory activity, but with fewer side effects.
Santhera’s application is supported by data from a 48-week Phase 2b trial (NCT03439670) called VISION-DMD.
The trial enrolled 121 boys with DMD who were randomly assigned to receive daily doses of vamorolone at 2 or 6 mg/kg, prednisone at 0.75 mg/kg, or a placebo.
Vamorolone significantly improved Time to Stand (TTSTAND) after 24 weeks, meeting the trial’s primary goal. TTSTAND evaluates muscle strength by measuring the speed at which a patient stands up from lying down.
The therapy also led to significant improvement in the six-minute walk test (6MWT), which assesses the distance walked in six minutes. Vamorolone also was significantly superior to a placebo in the NorthStar Ambulatory Assessment total score for motor function and the time to climb four stairs.
The application also was supported by results from three open-label studies wherein vamorolone was given at the same daily doses for 30 months (2.5 years). Collectively, these studies showed vamorolone didn’t stunt growth and had fewer side effects than those typical with corticosteroid use.
The Phase 2a trial VBP15-002 (NCT02760264) and its extension study VBP15-003 (NCT02760277) showed improved motor function in boys with DMD. A similar improvement was observed in the Phase 2 VBP15-003 trial (NCT02760277) and its two-year extension study, VBP15-LTE (NCT03038399).
Santhera plans to submit a new drug application for vamorolone in the U.S. this year. The treatment earned an orphan drug designation in the U.S. and Europe, as well as fast track and rare pediatric disease designations in the U.S. It also was granted the promising innovative medicine designation in the U.K. for DMD.
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