The FDA has approved a new drug from Roivant and partner Priovant Therapeutics for the treatment of a rare skin and muscle condition for use in the US.
Lisraya (brepocitinib), to be taken as a once daily pill, will now be available to patients with dermatomyositis (DM), a systemic autoimmune condition that causes painful skin rashes and legions, progressive muscle weakness and can severely affect quality of life if left untreated.
Brepocitinib is a first-in-class TYK2/JAK1 inhibitor, which works by blocking harmful inflammatory proteins, and is currently the first and only targeted therapy approved for dermatomyositis.
The drug performed well in a late-stage trial last year, showing improvement in both skin and muscle symptoms in DM patients, reducing or stopping the need for steroids that are commonly used as a treatment for the disease but can cause long-term side effects.
Based on the myositis Total Improvement Score, benefits were seen as early as 4 weeks into the trial, increased over time and were sustained until the end of the study, with brepocitinib achieved a final mean total improvement score of 46.5 after 52 weeks, compared with 31.2 for placebo.
The drug’s approval marks significant progress for treatment of the disease, for which there are no currently other specific treatments and patients have had to rely on non-targeted drugs.
Ben Zimmer, Chief Executive Officer of Priovant, said: “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.”
Ruth Ann Vleugels, MD, MPH, MBA, a leading expert in autoimmune connective tissue diseases and emerging therapies, said: “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.”
“The approval of Lisraya (brepocitinib) marks a turning point for patients living with dermatomyositis,” she added.
Following the approval announcement, Roivant shares closed up 2% on Thursday, putting its market value at roughly $27.1bn. That valuation has risen dramatically over the past year.
Analysts predict annual sales of more than $2 billion for brepocitinib in dermatomyositis alone by the early 2030s.
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