FDA Clears First Targeted Dermatomyositis Treatment From Roivant

Roivant Sciences Ltd (NASDAQ:ROIV) on Thursday secured U.S. Food and Drug Administration approval for LISRAYA (brepocitinib) 30 mg, a once-daily oral medication for adults living with dermatomyositis.

The regulatory decision brings the first and only targeted treatment to market for this rare systemic autoimmune disorder.

Physicians can prescribe the therapy as a standalone option or alongside existing non-targeted therapies, without restrictions regarding prior treatment history or baseline disease severity.

FDA Clearance Expands Dermatomyositis Treatment Options

Dermatomyositis causes progressive muscle weakness, chronic steroid reliance, and painful skin lesions sensitive to light and touch.

As a first-in-class TYK2/JAK1 inhibitor, LISRAYA provides broad clinical benefits, including improved muscle strength, skin clearance, enhanced physical function, and reduced heavy steroid use.

Phase 3 VALOR Trial Highlights Steroid Reduction

Approval stems from the Phase 3 VALOR study, the largest clinical trial completed in dermatomyositis.

Participants receiving LISRAYA showed statistically meaningful gains on the myositis Total Improvement Score by Week 4 and maintained progress through 52 weeks.

By study completion, 55% of LISRAYA patients achieved moderate or better disease improvement while requiring minimal to no steroids, compared with 30% of placebo participants.

Additionally, among patients using high-dose oral corticosteroids at baseline, 62% on LISRAYA reduced their dosage to minimal levels, compared with 38% on placebo. Furthermore, 45% of LISRAYA patients eliminated steroid usage, compared to 29% in the placebo arm.

Patient-Reported Outcomes Confirm Daily Functional Benefits

In addition to objective measures of muscle strength and skin symptoms, patients taking LISRAYA reported over fourfold greater improvement in overall disease activity than placebo recipients.

LISRAYA patients also registered meaningful gains in daily functional tasks such as climbing stairs and running errands, whereas placebo patients suffered functional decline.

ROIV Price Action: Roivant Sciences shares were down 1.37% at $34.23 at the time of publication on Monday, according to Benzinga Pro data.

Roivant-Photo by T. Schneider via Shutterstock