The U.S. Food and Drug Administration approved Lisraya (brepocitinib) tablets for the treatment of dermatomyositis in adults. Lisraya is the first FDA‑approved oral therapy specifically indicated for this rare autoimmune disorder, offering an alternative to treatments repurposed from other diseases. The approval was granted to Priovant Therapeutics Inc.
Lisraya is administered as a once‑daily oral tablet and functions as a JAK/TYK2 inhibitor. By inhibiting JAK signaling pathways, which are central to immune and inflammatory responses, brepocitinib aims to reduce the inflammatory processes that cause muscle damage and characteristic skin manifestations in dermatomyositis.
Efficacy and safety were evaluated in a phase 3, randomized, double‑blind, multicenter, placebo‑controlled study (clinicaltrials.gov identifier NCT05437263). The trial enrolled 241 adults with dermatomyositis who were randomized to receive either brepocitinib 30 mg once daily, brepocitinib 15 mg once daily, or placebo for a 52‑week treatment period.
The study used the Total Improvement Score (TIS) at week 52 as a principal measure to assess clinical response. TIS is a standardized composite that tracks changes across six domains: muscle strength, physical function, skin and other disease activity, muscle enzymes, and both physician and patient assessments of overall health.
Participants treated with Lisraya 30 mg once daily achieved a higher average Total Improvement Score (TIS) at week 52 compared with placebo, indicating greater overall clinical improvement and disease control. In addition to the improved composite score, patients on the 30 mg dose demonstrated benefits in specific domains, including physical function and skin disease activity. The 30 mg group was also more likely to reduce corticosteroid use by week 48 compared with placebo, suggesting potential steroid‑sparing effects in some patients.
Details on numerical TIS differences, responder rates, statistical testing, or outcomes for the 15 mg dose were not reported in the source beyond the summary that 30 mg produced higher average TIS and that other improvements were observed.
The most commonly reported adverse reactions with Lisraya in the clinical study included upper respiratory tract infection, headache, fatigue, urinary tract infection, and nausea. Treatment discontinuation due to adverse reactions occurred in 6% of participants treated with Lisraya 30 mg compared with 11% of those given placebo in the trial.
Lisraya carries a boxed warning that highlights the risks of serious infections, increased all‑cause mortality, malignancies, major adverse cardiovascular events (MACE), and thrombosis. Clinicians should consider these risks when evaluating patients for treatment with Lisraya and monitor patients according to current standards for JAK‑inhibitor class safety surveillance.
The FDA granted Lisraya both Orphan Drug designation and Priority Review. Orphan Drug status recognizes the treatment of a rare condition, and Priority Review indicates the FDA considered the application to address an unmet medical need.
The approval was assigned to Priovant Therapeutics Inc.
This approval provides clinicians and adults living with dermatomyositis an FDA‑approved, evidence‑based oral treatment option where previously management often relied on therapies intended for other conditions. The phase 3 data supporting approval showed meaningful improvements in a validated composite outcome (TIS), physical function, skin disease activity, and reduced corticosteroid use for patients receiving the 30 mg dose.
Given the boxed warning for serious infections, increased mortality risk, malignancy, MACE, and thrombosis, prescribers should weigh potential benefits against known safety risks and follow recommended monitoring for patients treated with Lisraya. Specific patient selection criteria, dose adjustments, lab monitoring schedules, and recommendations for managing adverse events were not detailed in the source and should be obtained from the full prescribing information.
This summary is based on the FDA press release announcing the approval of Lisraya (brepocitinib) for adult dermatomyositis and the referenced phase 3 study (NCT05437263). The FDA release includes statements from the agency and trial highlights; full trial results and the product labeling contain additional details not included in the press announcement.