FDA approves Priovant’s oral treatment for dermatomyositis
Key Takeaways
- FDA approval: Priovant received FDA approval for Lisraya (brepocitinib), an oral therapy for adults with dermatomyositis
- Phase 3 evidence: Over two-thirds (69%) of Lisraya patients showed a moderate or major improvement in their overall dermatomyositis symptoms after one year, compared to 47% taking placebo
- Next indication: Brepocitinib, a first-in-class TYK2/JAK1 inhibitor, is also being evaluated in a Phase 3 program in non-infectious uveitis, a Phase 3 program in cutaneous sarcoidosis, and a Phase 2b/3 program in lichen planopilaris.
Durham-based Priovant Therapeutics has received U.S. Food and Drug Administration (FDA) approval for Lisraya (brepocitinib) 30 mg for the treatment of adults with dermatomyositis. Lisraya is a first-in-class TYK2/JAK1 inhibitor taken orally once a day.
Dermatomyositis is a rare systemic autoimmune disease that can cause progressive muscle weakness and skin rashes, sometimes leading to serious complications such as difficulty swallowing, lung issues, or skin damage.
Although corticosteroids and immunosuppressants can help manage symptoms, they often come with significant side effects and don’t fully address the disease.
“The depth of clinical development talent and academic medicine in this region helped enable the company to take brepocitinib from a first-in-indication hypothesis to Lisraya, the first oral therapy ever approved for dermatomyositis,” said Ben Zimmer, CEO of Priovant. “The team here in North Carolina is what makes that work possible.”
The FDA approval of Lisraya follows the agency’s prior granting of Priority Review and Orphan Drug Designation.
The depth of clinical development talent and academic medicine in this region helped enable the company to take brepocitinib from a first-in-indication hypothesis to Lisraya, the first oral therapy ever approved for dermatomyositis.
— Ben Zimmer, Priovant CEO
’Phase 3 trial results
According to the company, the treatment provides a wide array of benefits for adults with dermatomyositis, including improvements in skin disease, muscle strength, physical function and overall disease burden, alongside substantial steroid-sparing benefit.
“(The FDA 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,” said Nikolay Nikolov, M.D., Director of the Office of Immunology and Inflammation in the FDA’s Center for Drug Evaluation and Research.
The approval follows the Phase 3 VALOR trial, one of the largest dermatomyositis trials ever conducted. The clinical study, published in the New England Journal of Medicine, showed that over two-thirds (69%) of Lisraya patients showed a moderate or major improvement in their overall dermatomyositis symptoms after one year (based on Total Improvement Score), compared to 47% taking placebo. Skin-specific results were also published in JAMA Dermatology.
“The FDA approval of LISRAYA marks a historic moment for the dermatomyositis community and reflects years of extraordinary work and sacrifice by the team at Priovant, our partners, and, above all, the investigators and patients who participated in the brepocitinib development program,” said Zimmer. “I am thrilled that adults with dermatomyositis finally have a fundamentally new treatment option – one specifically designed to target the biology of their disease and shown to meaningfully improve how patients feel and function in their daily lives.”
Brepocitinib, a first-in-class TYK2/JAK1 inhibitor, is also being evaluated in a Phase 3 program for non-infectious uveitis, a Phase 3 program for cutaneous sarcoidosis, and a Phase 2b/3 program for lichen planopilaris.
Priovant Therapeutics was founded in 2021 through a partnership between Roivant Sciences and Pfizer. The company was created around brepocitinib, a drug previously in development at Pfizer and now Priovant’s lead molecule.