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First Oral Option for Dermatomyositis Gets FDA Green Light
The approval gives adults with dermatomyositis a targeted oral option after a phase 3 trial showed better disease control and lower steroid use.
On Thursday, the U.S. Food and Drug Administration approved Lisraya, developed by Roivant and Priovant Therapeutics, as the first targeted oral therapy for dermatomyositis, a rare autoimmune disease causing muscle weakness and skin rashes.
Patients with dermatomyositis, which affects between 40,000 and 70,000 people in the U.S., have historically relied on high doses of steroids and other non-specific immune-suppressing drugs, according to Roivant.
The drug, a TYK2/JAK1 inhibitor, demonstrated efficacy in a phase 3 trial of 241 adults, with patients receiving 30 mg achieving a mean Total Improvement Score of 46.5 at 52 weeks.
Data showed 62% of patients discontinued or tapered steroids, though the treatment carries a boxed warning for serious infections, malignancies, and major adverse cardiovascular events.
Dr. Nikolay Nikolov of the FDA called the approval "a meaningful step forward," while the Muscular Dystrophy Association noted the therapy offers a new alternative for patients, with the drug immediately available in the U.S.