FDA approves first targeted oral drug for rare dermatomyositis; developed by former Pfizer team

The U.S. Food and Drug Administration (FDA) has approved Ryslaya, a treatment for rare dermatomyositis.

According to BioPharma Dive on the 27th (local time), the FDA approved Pfizer-discovered brepocitinib for sale under the name Ryslaya. Ryslaya is the first targeted oral treatment for patients with dermatomyositis, which causes muscle weakness and skin lesions.

The drug was licensed from Pfizer and developed by Priovant Therapeutics, a subsidiary of Roivant Sciences. Priovant has been studying Ryslaya in dermatomyositis and other rare diseases, including inflammatory eye and skin conditions.

Roivant CEO Matt Gline said in an interview with BioPharma Dive, “We chose markets with less competition and potentially faster approval pathways.” He added, “These are markets with high unmet need and a good fit for the drug’s biology.”

Ryslaya is taken once daily and targets the TYK2 and JAK1 proteins. An estimated 34,000 people in the U.S. have dermatomyositis, which has primarily been managed with steroids and immunoglobulin infusions.

In a phase 3 trial, patients who took a 30㎎ dose for 1 year had better scores on measures of muscle strength and function, skin health and other outcomes. Patients taking Ryslaya were also more likely to reduce or discontinue steroid use.

Additional data published this week in JAMA also showed improvement in skin symptoms. After 1 year of treatment, about three-quarters of patients had a clinically meaningful improvement in itching, far exceeding the 33% rate in the placebo group.

Nikolay Nikolov, director of the FDA’s Division of Immunology and Inflammation, said, “People with dermatomyositis have long faced substantial unmet need for effective treatments.” He added, “This approval is a meaningful step forward in providing an oral treatment to help manage this rare and debilitating disease.”

Common side effects of Ryslaya included infections, headache, fatigue, nausea and diarrhea. Priovant said Ryslaya’s safety profile is “consistent” with that of other JAK inhibitors.

Ryslaya’s prescribing information includes the same type of “boxed warning” as other JAK blockers. David Risinger, an analyst at Leerink Partners, said, “There were no major surprises in the label, and all the important secondary endpoints were included.”