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FDA approval · Priovant Therapeutics · Rheumatology

Lisrayabrepocitinib

FDA approves the first oral treatment for dermatomyositis in adults.

CompareFDA press release
FDA approval · New moleculeFirst oral treatment for dermatomyositis50-second read

Lisrayabrepocitinib

Priovant Therapeutics · Janus kinase (JAK/TYK2) inhibitor · Rheumatology

Bottom line for your patients

Adults with dermatomyositis now have an FDA-approved oral therapy that improved disease activity scores over placebo at one year. It carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis.

Drug brief
Drug IQ
Who it is for
Adults with dermatomyositis.
What changes
FDA notes that patients have had limited options and often relied on therapies meant for other diseases. The pivotal study compared brepocitinib 30 mg and 15 mg once daily with placebo.
The evidence
Participants treated with Lisraya 30 mg had a higher average Total Improvement Score at week 52 than those who received placebo, with improvements in physical function and skin disease activity. The FDA press release does not give the score values.Placebo-controlled randomized trialNCT05437263 · Phase 3 · 241 participantsRandomized against placebo: shows that it works, not how it compares with existing treatments.
Watch for
Boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis. Common adverse reactions included upper respiratory tract infection, headache, fatigue, urinary tract infection, and nausea; discontinuation due to adverse reactions occurred in 6% on Lisraya 30 mg and 11% on placebo.
How it is given
OralOnce-daily oral tablet. The trial tested 30 mg and 15 mg, and efficacy is reported for 30 mg. The recommended dose is not stated in the FDA release.
What the approval tells you
New molecule. An active ingredient the FDA had not approved before.Priority review. The FDA aimed to decide in about 6 months instead of the standard 10. A faster clock, not a higher or lower evidence bar.Orphan drug. Developed for a condition affecting fewer than 200,000 people in the US. Trials are often smaller.

Clinical summary

Lisraya is a once-daily oral JAK/TYK2 inhibitor approved to treat dermatomyositis in adults. In a phase 3 placebo-controlled study of 241 adults, participants on Lisraya 30 mg had a higher average Total Improvement Score at week 52 than those on placebo and were more likely to reduce corticosteroid use by week 48. It carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis.

From the source 11

FDA press release

The FDA press release's own words, quoted exactly. Each line is what a claim on this page was checked against.

  1. Lisraya is the first FDA-approved oral treatment option for patients with dermatomyositis, addressing the high unmet need for this rare disease.

    Backs · First oral treatment for dermatomyositis

  2. The U.S. Food and Drug Administration has approved Lisraya (brepocitinib) tablets for the treatment of dermatomyositis in adults.

    Backs · Approval, adults

  3. Lisraya is a once-daily oral tablet that works as a Janus kinase (JAK/TYK2) inhibitor.

    Backs · Once-daily oral JAK/TYK2 inhibitor

View all 11 lines

  1. The efficacy and safety of Lisraya were evaluated in a phase 3 randomized, double-blind, multicenter, placebo-controlled study (NCT05437263) that included 241 adults with dermatomyositis.

    Backs · Phase 3, placebo-controlled, 241 adults

  2. Participants were randomized to receive Lisraya (brepocitinib) 30 mg once daily, brepocitinib 15 mg once daily or placebo for a 52-week treatment period.

    Backs · 30 mg and 15 mg once daily vs placebo, 52 weeks

  3. Participants treated with Lisraya 30 mg had a higher average TIS score (indicating greater clinical response and disease control) at week 52 compared to those who received a placebo.

    Backs · TIS at week 52

  4. They also showed improvements in physical function and skin disease activity and were more likely to reduce corticosteroid use by week 48.

    Backs · Corticosteroid reduction by week 48

  5. Discontinuation due to adverse reactions occurred in 6% of participants treated with Lisraya 30 mg, compared with 11% of those given a placebo.

    Backs · Discontinuation 6% vs 11%

  6. Lisraya carries a boxed warning for serious infections, increased all-cause mortality (a higher risk of death from any cause), malignancies (cancers), major adverse cardiovascular events (serious heart and blood vessel problems), and thrombosis (blood clots).

    Backs · Boxed warning

  7. The FDA granted Lisraya Orphan Drug and Priority Review designations.

    Backs · Orphan Drug and Priority Review

  8. “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, M.D., Director of the Office of Immunology and Inflammation in the FDA’s Center for Drug Evaluation and Research.

    Backs · Limited options

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