FDA approves Lisraya, first targeted therapy for dermatomyositis

Overview

Updated Sep 16

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 is now on sale in the US.

Until now, doctors managed dermatomyositis with broad immunosuppressants and steroids that carry long-term side effects. In the 241-patient VALOR trial, Lisraya cut steroid use, and Leerink analysts project $1.8 billion in worldwide sales by 2032. Roivant, Priovant's parent company, is running late-stage trials of the drug in three more diseases.

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.

$1.8B

Projected 2032 worldwide dermatomyositis sales

Leerink forecast, cited by BioSpace. The same analysts see $4.2 billion in 2032 across all indications.

Voices

Curated perspectives — historical figures and your fellow readers.

Ever wondered what historical figures would say about today's headlines?

People Involved

Ben Zimmer

Chief executive of Priovant Therapeutics

Leading Lisraya's US launch

Matt Gline

Chief executive of Roivant Sciences

Overseeing brepocitinib's expansion beyond dermatomyositis

Organizations Involved

Priovant Therapeutics

Biopharmaceutical company

Developer and marketer of Lisraya

Biopharmaceutical company that developed Lisraya (brepocitinib) for dermatomyositis.

Roivant Sciences Ltd.

Biopharmaceutical holding company

Parent company of Priovant; trades on Nasdaq as ROIV

Holding company that builds and operates focused biotech subsidiaries, including Priovant.

U.S. Food and Drug Administration

Federal regulatory agency

Approved Lisraya after Priority Review and Orphan Drug Designation

US agency that regulates drug approvals and drug safety.

Muscular Dystrophy Association

Nonprofit disease research and advocacy organization

Welcomed the approval

Advocacy group representing people with muscle diseases including dermatomyositis.

Timeline

2021 August 2026

6 events Latest: August 28th, 2026 · 1 month ago

Tap a bar to jump to that date

August 2026
Roivant investor call scheduled
Latest Corporate Update

Roivant will host an investor call at 8:00 a.m. ET to discuss the approval and launch.
August 28th, 2026 • 8:00 AM
Leerink projects $1.8 billion in Lisraya sales by 2032
Market Analysis

Leerink analysts expect a 'slow and steady' launch, with $49 million in 2027 worldwide dermatomyositis sales growing to $1.8 billion by 2032, and $4.2 billion that year across all indications.
August 28th, 2026
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.
August 27th, 2026
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.
August 27th, 2026
Roivant says brepocitinib is in late-stage trials for three more diseases
Corporate Update

Roivant CEO Matt Gline called the dermatomyositis approval 'the first of many' for brepocitinib and said the company is advancing late-stage programs in non-infectious uveitis, cutaneous sarcoidosis, and lichen planopilaris.
August 27th, 2026
2021
Pfizer licenses brepocitinib to Priovant
Licensing

Pfizer licensed brepocitinib to Priovant Therapeutics, a joint company it created with Roivant Sciences.
2021

Scenarios

Lisraya becomes the standard of care for dermatomyositis

Possible Resolves by Feb 15, 2028

Discussed by: Sell-side analysts, including the analyst Cheng cited by Reuters, who forecasts more than $2 billion in peak US annual sales

Real-world prescribing follows the VALOR results, and the once-daily pill with a steroid-sparing profile becomes the default therapy for newly diagnosed patients. Insurance coverage and the $0-per-month copay program for eligible patients drive rapid uptake. Sales climb toward analyst forecasts by the early 2030s.

FDA adds safety restrictions to Lisraya's label

Possible Resolves by End of 2027

Discussed by: Regulatory observers who note JAK inhibitors carry boxed warnings for infections, cardiovascular events, and malignancy

If post-approval studies or real-world reports surface risks, the FDA could add boxed warnings or restrict Lisraya to certain patient populations. That would echo the safety reviews that reshaped earlier JAK inhibitor labels, and would dampen use despite positive efficacy data.

Lisraya stays a niche therapy after slow launch

Possible Resolves by Q3 2027

Discussed by: Skeptics who note the small patient population and the existing IVIG option, Octagam 10%

With fewer than 5,000 US patients, the addressable market is small. Specialty pharmacy distribution and competition from the only other approved modern therapy could slow adoption. If first-year sales disappoint, analysts cut peak forecasts and the drug remains a niche option.

Brepocitinib wins approval in a second disease

Possible Resolves by End of 2027

Discussed by: Roivant leadership; Leerink analysts who model $4.2 billion in 2032 sales across all indications

Roivant is running late-stage trials of brepocitinib in non-infectious uveitis, cutaneous sarcoidosis, and lichen planopilaris. An approval in any of them would broaden the drug's market beyond the roughly 5,000 US dermatomyositis patients and support the cross-indication sales forecast.

Historical Context

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

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.

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.