FDA Approves Lisraya for Adult Dermatomyositis

Overview

• The FDA has approved Priovant Therapeutics’ Lisraya (brepocitinib) to treat dermatomyositis in adults, making it the first pill authorized specifically for this rare muscle-and-skin autoimmune disease.
• Approval was based on a phase 3, randomized, double-blind, placebo-controlled study of 241 adults that used the Total Improvement Score (TIS) at week 52 as the primary endpoint.
• Patients taking 30 mg daily reached a mean TIS of 46.5 at 52 weeks versus 31.2 for placebo, with signs of improvement reported as early as week four and gains in both skin and muscle function.
• Priovant and partner Roivant launched the drug commercially with a 30-day list price reported at $35,000 and have started filling prescriptions, but analysts warn that pricing, patient identification, and a small eligible population create major uptake uncertainty.
• The approval could change care for people long treated with high-dose steroids or IV immunoglobulin, but clinicians will need to weigh the drug’s benefits against known JAK/TYK2 class safety concerns and the need for monitoring.