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Getting the latest healthcare news for you
Getting the latest healthcare news for you
A long-awaited first for a rare disease: the FDA has approved brepocitinib (Lisraya), the first oral therapy specifically indicated for dermatomyositis in adults. The once-daily JAK inhibitor outperformed placebo in a landmark phase 3 trial, offering a targeted alternative to the cumbersome, often toxic treatments patients have relied on for years. The drug is already available in the US.
A long-awaited first for a rare disease
The FDA has approved brepocitinib (Lisraya) — the first oral drug specifically indicated for dermatomyositis in adults. Made by Priovant Therapeutics, this once-daily tablet works by selectively blocking JAK pathways to reduce the harmful inflammation that drives this rare autoimmune disease, which causes muscle weakness and skin rashes. Until now, patients had to rely on treatments borrowed from other diseases — like disease-modifying antirheumatic drugs and IV immune globulin — that are difficult to administer, offer limited efficacy, and carry significant toxicity.
The approval is backed by the VALOR trial, a phase 3, double-blind, randomized, placebo-controlled study published in The New England Journal of Medicine. Patients on the 30-mg dose showed meaningful clinical improvement, and the drug outperformed placebo across all nine key secondary endpoints.
By the Numbers
Why it matters: Dermatomyositis patients have faced a significant treatment gap for decades. Brepocitinib gives clinicians a targeted, oral option that could allow earlier intervention and help patients avoid the harsh side effects of traditional therapies.
