The US Food and Drug Administration approved brepocitinib (Lisraya) tablets for adults with dermatomyositis, providing the first US Food and Drug Administration-approved oral treatment option indicated for the disease, according to an agency press release.
Dermatomyositis is a rare autoimmune condition affecting the muscles and skin that can cause chronic inflammation, progressive muscle weakness, and skin rashes. Brepocitinib is a once-daily oral Janus kinase and tyrosine kinase 2 inhibitor that blocks pathways involved in immune and inflammatory responses.
“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.
The efficacy and safety of brepocitinib were evaluated in a phase 3 randomized, double-blind, multicenter, placebo-controlled study involving 241 adults with dermatomyositis. Participants were randomly assigned to receive brepocitinib 30 mg once daily, brepocitinib 15 mg once daily, or placebo for 52 weeks.
The study evaluated Total Improvement Score at week 52, a standardized measure assessing changes in 6 areas: muscle strength, physical function, skin and other disease activity, muscle enzymes, and physician and patient assessments of overall health.
At week 52, participants who received brepocitinib 30 mg had a higher average Total Improvement Score compared with those who received placebo. Participants receiving the 30-mg dose also showed improvements in physical function and skin disease activity and were more likely to reduce corticosteroid use by week 48.
Some of the most common adverse reactions were upper respiratory tract infection, headache, fatigue, urinary tract infection, and nausea. Adverse reactions led to treatment discontinuation in 6% of participants who received brepocitinib 30 mg compared with 11% of those who received placebo.
Brepocitinib carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis. The FDA granted brepocitinib Orphan Drug and Priority Review designations. The approval was granted to Priovant Therapeutics Inc.
Source: US Food and Drug Administration
