DURHAM, N.C. — March 3, 2026 — Leads & Copy — The U.S. Food and Drug Administration (FDA) has accepted Priovant Therapeutics’ New Drug Application (NDA) for brepocitinib for the treatment of dermatomyositis (DM) and granted it Priority Review. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date in the third quarter of calendar year 2026.
Priovant anticipates launching brepocitinib in the United States by the end of September 2026.
Priority Review is given to applications for drugs that, if approved, would significantly improve the safety or effectiveness of treating a serious condition. The Priority Review designation was supported by the significant unmet medical need in dermatomyositis and results from the Phase 3 VALOR study, which evaluated brepocitinib in DM.
The VALOR study (N=241) was the longest and largest interventional dermatomyositis trial to date and the first positive 52-week placebo-controlled study in DM.
Dr. Ruth Ann Vleugels, M.D., M.P.H., M.B.A., from Mass General Brigham and Harvard Medical School, noted the decades-long wait for innovative therapeutics targeting the underlying biology of dermatomyositis. She expressed excitement about the potential FDA approval of the first targeted therapy for the disease.
Dr. Vleugels added that the Phase 3 data suggests the therapy could significantly improve patients’ quality of life and function with a once-daily oral therapy. She also emphasized the urgent need for better treatment options for dermatomyositis patients.
Dermatomyositis patients often experience a significant disease burden from muscle and skin issues, and they frequently rely on chronic high-dose steroids. Accumulated organ damage from uncontrolled chronic inflammation and the burdens of chronic steroid dependency contribute to high rates of comorbidities among DM patients on current standard of care.
According to Ben Zimmer, CEO of Priovant, the acceptance of the NDA represents meaningful progress towards bringing a potentially transformational therapy to dermatomyositis patients. Zimmer said the company is committed to working closely with the FDA to make the drug available as quickly as possible.
The VALOR study was a global Phase 3 trial involving 241 subjects with dermatomyositis across 90 sites. Participants were randomized to brepocitinib 30 mg, brepocitinib 15 mg, or placebo. The 30 mg dose showed statistically significant and clinically meaningful improvement compared to placebo on the primary endpoint of myositis Total Improvement Score (TIS) at Week 52. The company reported that benefit compared to placebo was seen as early as Week 4 and sustained throughout the one-year treatment period.
Priovant reported that the 30 mg dose also demonstrated statistically significant and clinically meaningful improvement compared to placebo on all nine Key Secondary endpoints evaluated, including measurements of skin disease, muscle disease, and steroid sparing. Over two-thirds of patients on brepocitinib 30mg achieved a Total Improvement Score of at least 40 (TIS40), and more than half achieved this threshold while also achieving steroid dependency of ≤2.5 mg/day.
The VALOR trial included a broad DM population, including patients with a history of benign or malignant neoplasm and multiple cardiovascular risk factors. Serious infections were more frequent in the brepocitinib 30 mg group compared to placebo; these events resolved with medical management, and brepocitinib treatment was completed in most cases. New or recurrent malignancy, cardiovascular events, and thromboembolic events occurred more frequently in the placebo arm than the brepocitinib 30 mg arm.
The brepocitinib safety database, encompassing over 2,000 patients and subjects across all studies, suggests a safety profile similar to approved JAK and TYK2 inhibitors.
Priovant Therapeutics is focused on developing therapies for autoimmune diseases with limited treatment options. Their lead asset, brepocitinib, is a dual selective inhibitor of TYK2 and JAK1. It suppresses key cytokines linked to autoimmunity—including type I IFN, type II IFN, IL-6, IL-12, and IL-23—with a single, targeted, once-daily oral therapy.
Brepocitinib recently generated positive Phase 3 data in dermatomyositis, and its New Drug Application is under FDA review. Brepocitinib is also being evaluated in a Phase 3 program in non-infectious uveitis and recently generated positive Phase 2 data in cutaneous sarcoidosis, with a Phase 3 study to begin in calendar year 2026. Priovant Therapeutics is a Roivant (Nasdaq: ROIV) company.
Source: Priovant Therapeutics
