Santhera Pharmaceuticals Holding AG: Santhera and ReveraGen Announce New 2.5-year Treatment Data with Vamorolone in Duchenne Muscular Dystrophy
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A sequence of studies investigated the safety, tolerability and long-term treatment benefit with vamorolone and provide open-label data in a total of 46 patients with DMD, aged 4 to up to 10 years (start/end of treatment), of which 41 (89%) completed a 2.5 years treatment period. 48 participants initially completed a two-week multiple ascending dose trial VBP15-002 [1], 46 of whom entered the open-label 6-month extension VBP15-003 [2], covering a dose range of vamorolone of 0.25 to 6.0 mg/kg/day. All of the 46 patients completing the latter study entered the 24-month long-term extension study VBP15-LTE [3] where all patients eventually received doses of vamorolone of 2.0 to 6.0 mg/kg/day. The total exposure to vamorolone treatment in these studies was 113 patient years. The most commonly administered dose of vamorolone was 6.0 mg/kg/day. The studies, conducted by the Cooperative International Neuromuscular Research Group (CINRG), evaluated drug-related effects of vamorolone on timed motor function outcomes and corticosteroid-associated safety concerns.