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FDA approves Lisraya, first targeted therapy for dermatomyositis

FDA approves Lisraya, first targeted therapy for dermatomyositis

New Capabilities

Once-daily pill for rare autoimmune disease cuts steroid dependence

Today: Roivant investor call scheduled

Overview

Updated 2 hours ago

The FDA approved Lisraya on August 27, 2026, clearing the first targeted therapy ever approved for dermatomyositis, a rare autoimmune disease that inflames skin and weakens muscles. The once-daily pill showed gains in skin disease, muscle strength, and physical function within four weeks.

Until now, doctors managed dermatomyositis with broad immunosuppressants and steroids that carry long-term side effects. Lisraya's steroid-sparing results could change how the roughly 5,000 Americans with the condition are treated.

Why it matters

Dermatomyositis patients finally have a pill that targets their disease's cause instead of suppressing the whole immune system, and it cuts steroid dependence.

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Key Indicators

30 mg
Lisraya daily dose
Lisraya is taken once daily as a 30 mg pill.
55% vs 30%
Patients reaching moderate improvement plus minimal or no steroid use
Measured at 52 weeks in the Phase 3 VALOR trial: Lisraya versus placebo.
62% vs 38%
Patients tapering to minimal or no steroid use
Among those on at least 7.5 mg/day of prednisone-equivalent at baseline, Lisraya versus placebo.
5,000
People in the US with dermatomyositis
Estimate from the National Institutes of Health.
$2B
Projected peak US annual sales by early 2030s
Forecast by the analyst Cheng, cited by Reuters, for Lisraya in dermatomyositis alone.

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People Involved

Organizations Involved

Timeline

January 2021 August 2026

4 events Latest: Today
Tap a bar to jump to that date
  1. Roivant investor call scheduled

    Today Corporate Update

    Roivant will host an investor call at 8:00 a.m. ET to discuss the approval and launch.

  2. FDA approves Lisraya for dermatomyositis

    Regulatory Approval

    FDA approved Lisraya (brepocitinib) 30 mg as the first targeted therapy for adults with dermatomyositis, following Priority Review and Orphan Drug Designation.

  3. Lisraya launches in the US

    Launch

    The drug is available immediately through specialty pharmacies; eligible patients may pay as little as $0 per month through the My Compass Support program.

  4. Pfizer licenses brepocitinib to Priovant

    Licensing

    Pfizer licensed brepocitinib to Priovant Therapeutics, a joint company it created with Roivant Sciences.

Historical Context

2 moments from history that rhyme with this story — and how they unfolded.

March 2007

Soliris (eculizumab) approval (2007)

The FDA approved Soliris, the first complement inhibitor, for paroxysmal nocturnal hemoglobinuria, a rare blood disease previously managed with transfusions and supportive care.

Then

Soliris became the standard of care for the disease and proved targeted therapies could command premium pricing in rare diseases.

Now

It set a commercial template for ultra-rare disease drugs with small patient populations and high per-patient costs.

Why this matters now

Like Soliris, Lisraya is a first-targeted-therapy for a rare disease with a small US population, testing how far mechanism-based drugs can reshape rare disease care.

November 2012

Xeljanz (tofacitinib) approval (2012)

The FDA approved Xeljanz, the first JAK inhibitor, for rheumatoid arthritis. It offered a daily pill targeting immune pathways instead of injected biologics or broad immunosuppression.

Then

Xeljanz transformed RA care and became a blockbuster, validating JAK inhibition as a treatment strategy.

Now

Post-marketing safety trials later linked higher doses to cardiovascular events and malignancy, prompting FDA boxed warnings and restricted use.

Why this matters now

Lisraya is a TYK2/JAK1 inhibitor, so it faces the same long-term safety scrutiny that reshaped Xeljanz's label.

Sources

(7)