Key Takeaways:
The U.S. Food and Drug Administration approved Roivant's LISRAYA (brepocitinib) 30 mg for adults with dermatomyositis, the first targeted therapy for the rare autoimmune disease.
"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, professor of dermatology at Harvard Medical School and founding director of the Autoimmune Skin Disease Center at Mass General Brigham, said.
The approval follows the Phase 3 VALOR trial, the largest dermatomyositis study ever conducted. In the 52-week study, 55 percent of patients on LISRAYA achieved moderate or better improvement on the myositis Total Improvement Score with minimal or no steroid use, versus 30 percent on placebo. Among patients taking at least 7.5 mg per day of prednisone-equivalent corticosteroids at baseline, 62 percent tapered to minimal or no steroid use by week 52, compared with 38 percent on placebo; 45 percent stopped corticosteroids entirely, versus 29 percent on placebo. Benefits appeared as early as week four and were sustained through the study's end.
Roivant shares rose 1.58 percent to $37.33 on the news. The approval gives Roivant its first commercial product and confirms the brepocitinib platform, which is also in late-stage development for non-infectious uveitis, cutaneous sarcoidosis, and lichen planopilaris.
LISRAYA, a first-in-class TYK2/JAK1 inhibitor taken once daily, is available immediately in the U.S. through a limited network of specialty pharmacies. Eligible patients may pay as little as zero dollars per month through the LISRAYA My Compass Support program, Priovant said.
The drug also improved patient-reported outcomes. Patients on LISRAYA reported more than four times the improvement in overall disease activity versus placebo, and achieved clinically meaningful gains on a measure of everyday function covering pain and daily activities such as getting dressed and climbing stairs, while placebo patients worsened.
The most common adverse reactions were upper respiratory tract infection, headache, fatigue, urinary tract infection, nausea, bronchitis, arthralgia, diarrhea, back pain, fall, influenza, and acne. LISRAYA carries a boxed warning for serious infections, mortality, malignancy, major adverse cardiovascular events, and thrombosis, and is not recommended for use with other JAK inhibitors, TYK2 inhibitors, or biologic disease-modifying antirheumatic drugs.
Primary results from VALOR were published in the New England Journal of Medicine in March, with skin-specific secondary endpoints in JAMA Dermatology in August. The FDA granted LISRAYA Priority Review and Orphan Drug Designation.
The approval hands Roivant a revenue-generating asset in a disease where patients have long relied on steroids and intravenous immunoglobulin, and opens a path for brepocitinib across multiple autoimmune indications. Roivant will host an investor call on Aug. 28 at 8 a.m. ET to discuss the launch and pipeline.
This article is for informational purposes only and does not constitute investment advice.
