FDA Approves Brepocitinib as First Oral Drug for Dermatomyositis

For the first time, adults with dermatomyositis have an FDA-approved oral medicine designed to target the disease itself rather than dampen the immune system broadly, after the agency cleared brepocitinib (Lisraya; Roivant) on the strength of phase 3 trial data showing durable improvement in muscle, skin, and overall disease activity alongside meaningful reductions in steroid use.^1,2

Dermatomyositis causes progressive muscle weakness alongside extensive, painful, and pruritic skin lesions, and it significantly impairs patients' quality of life through physical disability, cutaneous disfigurement, and dependency on chronic high-dose steroids.¹

“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,” 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, said in a statement.¹ “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.”

The Results of the Pivotal Trial

The approval rests on the phase 3 VALOR trial (NCT05437263), described by the sponsor as the largest dermatomyositis trial ever conducted, which randomized 241 adults 1:1:1 to once-daily brepocitinib 30 mg, brepocitinib 15 mg, or placebo over 52 weeks.^1,2 Brepocitinib is a once-daily tablet that works as a Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2) inhibitor, blocking immune signaling pathways implicated in the disease's pathogenesis.³

The 30-mg dose met the trial's primary end point, a composite Total Improvement Score (TIS) that tracks 6 domains including muscle strength, physical function, and skin and overall disease activity, and it also met all 9 key secondary end points.² Patients on brepocitinib 30 mg were more than 4 times as likely to report improvement in overall disease activity compared with those on placebo. Unlike the placebo group, patients on brepocitinib achieved clinically meaningful gains in everyday function, including pain and activities such as dressing, climbing stairs, and running errands. Primary results were published in the New England Journal of Medicine in March 2026.⁴

How Much Did the Drug Improve Skin Disease and Itch?

A secondary analysis of VALOR published August 26 in JAMA Dermatology drilled into cutaneous outcomes, which are a major but often underappreciated driver of morbidity in dermatomyositis.⁵ Among patients with moderate to severe skin disease at baseline, 45.7% of those on brepocitinib 30 mg achieved “clear” or “almost clear” skin by week 52, versus 21.8% on placebo, and 43.5% reached functional skin remission versus 20.8% on placebo.

Itch, often the most debilitating and treatment-refractory symptom of dermatomyositis, also responded quickly: among patients with at least moderate baseline pruritus, 54% achieved clinically meaningful itch reduction by week 4 on brepocitinib 30 mg, compared with just 9.5% on placebo, a gap that persisted through week 52 (74.0% vs 33.3%). Skin-related quality of life, measured by the Skindex-16 instrument, improved by an amount exceeding the threshold considered clinically meaningful as early as week 4.

Sparring Patients From Steroids

Dermatomyositis has long been managed with chronic high-dose corticosteroids, which carry their own cumulative toxicity. In the VALOR trial, 61.7% of patients on oral corticosteroids at baseline who were treated with brepocitinib 30 mg tapered to a prednisone-equivalent dose of 2.5 mg/day or less by week 52, compared with 34.4% on placebo, while 41.7% discontinued steroids altogether versus 23.4% on placebo.^1,5 More than half of brepocitinib-treated patients achieved both a clinically meaningful TIS response and minimal or no steroid use by the end of the study, compared with 30% on placebo.¹

Safety findings were consistent with the established profile of the JAK inhibitor class. Serious infections, most respiratory in nature, occurred more often with brepocitinib than placebo, while malignancies, major adverse cardiovascular events, and thromboembolic events were more frequent in the placebo arm.^1,5 Discontinuation due to adverse reactions occurred in 6% of patients on brepocitinib 30 mg, compared with 11% on placebo.³ Like other JAK inhibitors, brepocitinib carries a boxed warning for serious infections, increased all-cause mortality, malignancy, major adverse cardiovascular events, and thrombosis.

What Should Managed Care Stakeholders Watch For?

Brepocitinib’s launch puts a new specialty oral drug in front of payers who will need to establish coverage criteria for a rare disease population with few precedents for comparison. The manufacturer said brepocitinib will be distributed through a limited network of specialty pharmacies, and the company is offering a patient assistance program, My Compass Support, through which eligible patients may pay as little as $0 per month, along with help navigating insurance coverage.¹ However, the drug's list price was not disclosed in the announcement materials.

Patients with rare rheumatologic and autoimmune diseases frequently encounter prior authorization and step-therapy hurdles rooted in a historical lack of FDA-approved, on-label treatment options. In a 2022 interview with The American Journal of Managed Care®, Christopher Phillips, MD, a rheumatologist in Kentucky, described as forcing patients to wait weeks only to learn a payer wants a different treatment tried first.⁶

An ongoing 52-week open-label extension of VALOR is expected to generate longer-term data on the drug's use under more flexible background-therapy conditions.²

“For too long, patients with dermatomyositis have faced a significant unmet need for effective treatments, often relying on therapies meant for other diseases,” said Nikolay Nikolov, MD, director of the Office of Immunology and Inflammation in the FDA’s Center for Drug Evaluation and Research. “Today’s approval is a meaningful step forward, giving patients and their healthcare providers an approved oral therapy proven to help manage this rare and debilitating disease.”

References

1. Roivant announces FDA approval of Lisraya (brepocitinib) for adults with dermatomyositis; now available in the U.S. Roivant Sciences. News release. Roivant Sciences. August 27, 2026. Accessed August 27, 2026. https://investor.roivant.com/news-releases/news-release-details/roivant-announces-fda-approval-lisrayatm-brepocitinib-adults
2. JAMA Dermatology publishes skin-specific outcomes from phase 3 VALOR trial of brepocitinib in dermatomyositis. News release. Roivant Sciences. August 26, 2026. Accessed August 27, 2026. https://investor.roivant.com/news-releases/news-release-details/jama-dermatology-publishes-skin-specific-outcomes-phase-3-valor
3. FDA approves first oral drug indicated to treat dermatomyositis in adults. US Food and Drug Administration. August 26, 2026. Accessed August 27, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-drug-indicated-treat-dermatomyositis-adults
4. Vleugels RA, Paik JJ, Ventura IB, et al; VALOR Investigators. A phase 3 trial of brepocitinib in dermatomyositis. N Engl J Med. 2026;394(19):1883-1893. doi:10.1056/NEJMoa2503531
5. Mangold AR, Haemel A, Shahriari N, et al. Skin-specific outcomes of brepocitinib in patients with dermatomyositis: secondary analysis of a phase 3 randomized clinical trial. JAMA Dermatol. Published online August 26, 2026. doi:10.1001/jamadermatol.2026.3199
6. Inserro A. How prior authorization can impact patients with rare disease. AJMC^®. February 28, 2022. Accessed August 27, 2026. https://www.ajmc.com/view/how-prior-authorization-can-impact-patients-with-rare-disease