First Oral Option for Dermatomyositis Gets FDA Green Light

The FDA approved the first oral treatment option for the autoimmune disorder dermatomyositis.

Brepocitinib (Lisraya) is indicated for adults with the rare condition, which causes chronic inflammation, progressive muscle weakness, and skin rashes. The oral drug addresses a longstanding need for patients who have few treatment options for symptom control.

“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, of the FDA’s Center for Drug Evaluation and Research, in the agency’s press release. “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.”

A TYK2/JAK1 inhibitor, brepocitinib helps reduce inflammation known to damage skin and muscle tissue.

“Dermatomyositis affects nearly every aspect of a patient’s life, causing physical disability, disfiguring skin disease, pain, itch, and a profound loss of independence and sense of self,” said Ruth Ann Vleugels, MD, MPH, of Mass General Brigham and Harvard Medical School in Boston, in an announcement from drugmaker Priovant Therapeutics. “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. The approval of Lisraya marks a turning point for patients living with dermatomyositis.”

Support for the approval came from the phase III, placebo-controlled VALOR trial involving 241 patients with longstanding, treatment-resistant dermatomyositis. The trial met the primary endpoint of Total Improvement Score (TIS) at 52 weeks. TIS is a standardized clinical scoring tool that tracks changes across six disease-related domains to assess a patient’s overall improvement.

The primary analysis showed a mean TIS of 31.2 for placebo-treated patients, 37.5 for the lower of two doses of brepocitinib, and 46.5 for the higher dose of the TYK2/JAK1 inhibitor (P<0.001).

The data showed that 45% of patients treated with brepocitinib discontinued corticosteroids, and 62% discontinued or tapered to a minimal dose, as compared with 38% and 29% of placebo-treated patients. Brepocitinib was also associated with improvement in multiple patient-reported outcomes.

The incidence of adverse events (AEs) was similar across treatment groups (86-91%). The most common any-grade AEs in any group (≥5%) were upper respiratory tract infection, COVID, urinary tract infection, nausea, diarrhea, and headache.

Brepocitinib will be available immediately in the U.S., according to the company announcement.