FDA Approval of LISRAYA for Adults with Dermatomyositis – Roivant
Roivant announced that the FDA has approved LISRAYA (brepocitinib) 30 mg for the treatment of adults with dermatomyositis (DM).
LISRAYA is a pill taken once daily. DM is a rare systemic autoimmune disease characterized by progressive muscle weakness and extensive, painful, and pruritic skin lesions. DM significantly impairs patients’ quality of life through physical disability, pain from both muscle and skin disease, cutaneous disfigurement, skin sensitivity to light and touch, and high levels of dependency on chronic high-dose steroids.
“We are hopeful the approval of LISRAYA in DM will be the first of many for brepocitinib, and will provide an important new treatment option for these patients.” said Matt Gline, CEO of Roivant. “I’m proud of the work our teams have put in to get here, and we remain focused on advancing our late-stage programs in non-infectious uveitis, cutaneous sarcoidosis, and lichen planopilaris — all also diseases where patients have been waiting a long time for better options.”
LISRAYA, a first-in-class TYK2/JAK1 inhibitor, is the first and only targeted therapy approved for dermatomyositis. LISRAYA is proven to provide a wide array of efficacy benefits for adult DM patients, including improvements in skin disease, muscle strength, physical function, and overall disease burden, alongside substantial steroid-sparing benefit. LISRAYA can be prescribed by healthcare professionals in the United States effective immediately.
“Dermatomyositis affects nearly every aspect of a patient’s life, causing physical disability, disfiguring skin disease, pain, itch, and a profound loss of independence and sense of self,” said Ruth Ann Vleugels, MD, MPH, MBA, Heidi and Scott C. Schuster Distinguished Chair in Dermatology, Founding Director of the Autoimmune Skin Disease Center and Connective Tissue Disease Clinics at Mass General Brigham, and Professor of Dermatology at Harvard Medical School. “For many decades, the treatment of dermatomyositis has relied on chronic steroids, non-specific immunomodulators, and intravenous immunoglobulin—therapies not targeted to the underlying disease pathobiology. The approval of LISRAYA marks a turning point for patients living with dermatomyositis. For the first time, I am thrilled to be able to offer my patients a targeted, once-daily oral medicine that delivers meaningful benefit across muscle, skin, and overall disease activity while simultaneously reducing reliance on systemic corticosteroids.”
LISRAYA’s approval follows the landmark Phase 3 VALOR trial, the largest DM trial ever conducted.
In the VALOR trial, benefits on the primary endpoint, the myositis Total Improvement Score (a composite measure designed to capture improvement across multiple disease domains), were seen as early as Week 4, increased over time, and were sustained to the end of the 52-week study. Most patients treated with LISRAYA were able to achieve both moderate or better improvement on the Total Improvement Score and minimal or no steroid use by the end of the study (55%, compared to 30% on placebo), underscoring LISRAYA’s ability to simultaneously improve disease symptoms and reduce steroid dependency. Among patients receiving LISRAYA who were taking ≥7.5 mg/day (prednisone-equivalent) of oral corticosteroids at baseline, 62% tapered to minimal or no steroid use (≤2.5 mg/day) by the end of the 52-week study, compared with 38% on placebo; 45% came off corticosteroids entirely, compared with 29% on placebo.
LISRAYA also demonstrated benefit on independent measures of skin disease and muscle strength, and, critically, on endpoints that directly capture patients’ own experience living with dermatomyositis. When asked to rate the overall activity of their disease, patients receiving LISRAYA reported more than four times as much improvement as patients on placebo. On a measure of everyday function – including pain and core activities of daily living such as getting dressed, climbing stairs, and running errands – patients treated with LISRAYA achieved clinically meaningful improvement, while those receiving placebo worsened, highlighting LISRAYA’s ability to restore greater independence and personal agency to DM patients’ lives.
The most common adverse reactions for patients on LISRAYA in the VALOR trial were upper respiratory tract infection, headache, fatigue, urinary tract infection, nausea, bronchitis, arthralgia, diarrhea, back pain, fall, influenza, and acne.
Primary results from the VALOR trial were published in the New England Journal of Medicine in March 2026, with additional skin-specific secondary endpoints published in JAMA Dermatology in August 2026.
LISRAYA is indicated for the treatment of dermatomyositis in adults. It can be used for adult DM patients with no restrictions based on their level of disease activity, clinical presentation, or prior treatment experience. LISRAYA can be used as an alternative therapy or add-on therapy to non-targeted DM treatments used today, depending on specific patient needs. Notably, the Phase 3 VALOR study evaluated the efficacy and safety of LISRAYA across patients on a wide array of different combinations of background therapies, including no background therapy.
“Today’s approval of LISRAYA marks a historic moment for the dermatomyositis community and reflects years of extraordinary work and sacrifice by the team at Priovant, our partners, and, above all, the investigators and patients who participated in the brepocitinib development program,” said Ben Zimmer, Chief Executive Officer of Priovant. “I am thrilled that adults with dermatomyositis finally have a fundamentally new treatment option – one specifically designed to target the biology of their disease and shown to meaningfully improve how patients feel and function in their daily lives.”
FDA approval of LISRAYA follows the agency’s prior granting of Priority Review and Orphan Drug Designation. Priority Review is reserved for medicines that, if approved, would provide significant improvements in safety or efficacy for treatment of a serious condition.
Citation: A Phase 3 Trial of Brepocitinib in Dermatomyositis. Authors: Ruth Ann Vleugels, M.D., M.P.H., Julie J. Paik, M.D., M.H.S. et al. Published March 28, 2026 N Engl J Med 2026;394:1883-1893 DOI: 10.1056/NEJMoa2503531 VOL. 394 NO. 19
Skin-Specific Outcomes of Brepocitinib in Patients With Dermatomyositis. Secondary Analysis of a Phase 3 Randomized Clinical Trial. Authors: Aaron R. Mangold, MD; Anna Haemel, MD; Neda Shahriari, MD et al. JAMA Dermatol Published Online: August 26, 2026 doi: 10.1001/jamadermatol.2026.3199
