FDA Accepts and Grants Priority Review of NDA for Brepocitinib in Dermatomyositis

Priovant Therapeutics recently announced that the FDA has accepted its new drug application (NDA) for brepocitinib, a tyrosine kinase 2/Janus kinase 1 (TYK2/JAK1) inhibitor, for the treatment of dermatomyositis and granted the application priority review. The FDA has assigned a Prescription Drug User Fee Act target action date in the third quarter of 2026. If approved, Priovant Therapeutics anticipates a launch in the US at the end of September 2026.¹

In dermatomyositis, where there has been a significant unmet need for therapeutic advances, the priority review designation reflects both the disease’s morbidity and the clinical data from the phase 3 VALOR trial (NCT05437263).

Dermatomyositis is characterized by inflammatory myopathy with cutaneous involvement, frequently accompanied by interstitial lung disease, malignancy risk, and substantial steroid exposure. Persistent disease activity and cumulative organ damage contribute to functional impairment and comorbidities, demonstrating the need for targeted, steroid-sparing strategies.

“[Patients with] dermatomyositis and medical communities have been waiting for decades for novel innovative therapeutics that directly target the underlying disease biology, and it is incredibly exciting to have the finish line in sight for the potential first FDA approval of a targeted therapy for this debilitating disease,” Ruth Ann Vleugels, MD, MPH, MBA, said in the news release. Vleugels is the Heidi and Scott C. Schuster Distinguished Chair in Dermatology at Brigham and Women’s Hospital, director of the Autoimmune Skin Diseases Program and Connective Tissue Disease Clinics at Mass General Brigham, and program director of the Dermatology-Rheumatology Fellowship Program at Harvard Medical School in Boston, Massachusetts.

Supporting Data

The FDA’s decision was supported by results from the global phase 3 VALOR study, the largest and longest interventional dermatomyositis trial to date, and the first positive 52-week placebo-controlled study in this population. Conducted across 90 sites, participants were randomly assigned 1:1:1 to receive brepocitinib 30 mg, brepocitinib 15 mg, or placebo.

At week 52, brepocitinib 30 mg achieved statistically significant and clinically meaningful improvement vs placebo on the primary end point, the myositis Total Improvement Score (TIS), a composite of 6 core measures of disease activity. Separation from placebo was observed as early as week 4 and sustained throughout the double-blind period. The 30-mg dose also met all 9 key secondary end points, including measures of cutaneous disease, muscle strength, and steroid sparing.

More than two-thirds of patients receiving brepocitinib 30 mg achieved TIS40—twice the minimum clinically important difference—and more than half reached this threshold while tapering to less than or equal to 2.5 mg/day of steroids. Approximately 75% of participants entered VALOR on background steroids, with a mean baseline dose of 12.2 mg/day in the brepocitinib 30 mg arm and 11.3 mg/day in the placebo arm. Among these patients, 62% treated with brepocitinib 30 mg reduced their steroid dose to less than or equal to 2.5 mg/day by week 52 (vs 34% with placebo), and 42% were able to discontinue steroids entirely (vs 23% with placebo), reinforcing the steroid-sparing effect observed alongside improvements in global disease activity.^1,2

Safety

The VALOR cohort included a broad dermatomyositis population, encompassing patients with prior benign or malignant neoplasms and those with multiple cardiovascular risk factors. Serious infections were more frequent in the brepocitinib 30 mg arm compared with placebo, though events resolved with medical management, and treatment was completed in most cases. Notably, new or recurrent malignancies, cardiovascular events, and thromboembolic events occurred more frequently in the placebo arm than in the brepocitinib 30 mg arm.

Across studies, the brepocitinib safety database includes more than 2000 patients and suggests a profile similar to approved JAK and TYK2 inhibitors.

Looking Ahead

“The acceptance of our NDA for brepocitinib in dermatomyositis represents meaningful progress toward our goal of bringing a potentially transformational therapy to [patients with] dermatomyositis who urgently need better treatment options,” Ben Zimmer, CEO of Priovant, said in the news release. “We are committed to working closely with the FDA through their review to make this drug available for patients as quickly as possible.”

For dermatology clinicians managing complex connective tissue disease, the pending decision may signal a new, targeted option in a field previously dominated by broad immunosuppression.

“[Patients with] dermatomyositis…urgently need better treatment options. The brepocitinib phase 3 data suggest that this therapy has the potential to meaningfully improve these patients’ quality of life and function with a once-daily oral therapy. I am thrilled regarding this major step forward in our ability to care for our patients with dermatomyositis,” Vleugels said.

References

1. Priovant announces FDA acceptance and priority review of new drug application for brepocitinib in dermatomyositis. News release. Roviant Sciences. March 3, 2026. Accessed March 3, 2026. https://investor.roivant.com/news-releases/news-release-details/priovant-announces-fda-acceptance-and-priority-review-new-drug/
2. Roivant and Priovant announce positive phase 3 VALOR study results for brepocitinib in 52-week placebo-controlled trial in dermatomyositis (DM). News release. Roviant Sciences. September 17, 2025. Accessed March 3, 2026. https://investor.roivant.com/news-releases/news-release-details/roivant-and-priovant-announce-positive-phase-3-valor-study