Roivant Sciences Wins FDA Approval for LISRAYA in Dermatomyositis, Launches Immediately

Key Points

• FDA approval and immediate launch: Roivant’s subsidiary Priovant received FDA approval for LISRAYA (brepocitinib), a once-daily oral therapy for adults with dermatomyositis, and began commercial sales the same day.
• Clinical and pricing profile: The therapy is the first targeted and steroid-sparing treatment approved specifically for dermatomyositis. Its list price is $35,000 per 30-day supply, while Phase III data showed meaningful disease responses and substantial reductions in steroid use.
• Measured commercial outlook and expansion plans: Management expects “slow and steady” uptake as physicians and payers adapt, with patient assistance programs aimed at reducing out-of-pocket costs. Roivant plans to develop brepocitinib for additional indications, including non-infectious uveitis, cutaneous sarcoidosis and lichen planopilaris.

Roivant Sciences NASDAQ: ROIV said the U.S. Food and Drug Administration has approved LISRAYA, its once-daily oral therapy for adults with dermatomyositis, and that the drug has already entered commercial launch.

Matt Gline, Roivant’s CEO, called the approval a significant milestone for patients with dermatomyositis, a rare and heterogeneous inflammatory disease affecting skin, muscle and, in some cases, other organs. He said LISRAYA is the first targeted therapy approved for dermatomyositis, the first once-daily oral therapy approved for the condition, and the first treatment approved specifically with a steroid-sparing benefit in the disease.

The therapy, brepocitinib, is a TYK2/JAK1 inhibitor. Gline said it is also the first TYK2/JAK1 inhibitor approved for any indication. He noted that prior efforts to advance therapies including rituximab, Remicade and Enbrel in dermatomyositis-specific studies had not succeeded.

Commercial Launch Begins

Ben Zimmer, CEO of Priovant, said LISRAYA launched commercially on the same day as its approval, approximately one month ahead of the company’s initial plan. Prescribing is open, and Priovant has already received its first prescriptions, he said.

The drug carries a wholesale acquisition cost, or list price, of $35,000 for a 30-day supply. Gline estimated that the annual net value to the company for a patient receiving a full year of therapy could be in the low-to-mid-$300,000 range after accounting for compliance, gross-to-net adjustments and other factors. He cautioned that the estimate contains substantial uncertainty because LISRAYA is the first novel therapy to launch in this disease setting.

Priovant has established the My Compass Support program to assist patients with insurance navigation and financial assistance. Zimmer said the company expects many patients to have no out-of-pocket costs, while Gline said eligible patients could pay as little as $0 per month through copay support. The company is also using a limited network of specialty pharmacies intended to provide high-touch support for rare-disease patients.

Broad Label and Clinical Data

Zimmer said the FDA label provides a broad indication for adults with dermatomyositis, without restrictions based on clinical presentation, disease severity or prior treatment history. The label also does not restrict concomitant use with steroids, conventional synthetic disease-modifying antirheumatic drugs, or intravenous immunoglobulin, or IVIG.

According to Zimmer, the label includes efficacy information supporting improvements in skin disease, muscle strength, physical function and overall disease burden, as well as patient-reported and physician-reported outcomes. The company highlighted LISRAYA’s steroid-sparing findings, including reductions in steroid use compared with placebo and measures of the ability to improve disease while reducing steroid dependence.

The approval was supported by the Phase III VALOR study, which Gline described as the largest placebo-controlled Phase III trial conducted in dermatomyositis. He said the study met its primary endpoint and every ranked secondary endpoint. VALOR data were published in The New England Journal of Medicine, while skin-specific secondary endpoint results were recently published in JAMA Dermatology.

• Nearly 70% of patients receiving LISRAYA achieved a moderate response on the Total Improvement Score, or TIS, according to the company.
• Nearly 50% achieved a major response.
• Among patients starting on at least 7.5 milligrams per day of steroids, nearly two-thirds reduced steroid use to 2.5 milligrams or less, while nearly half discontinued steroids altogether by the end of the study.

Zimmer said treatment discontinuations due to adverse events occurred at nearly twice the rate in the placebo group as in the LISRAYA group in VALOR. The drug’s label includes safety warnings associated with the JAK inhibitor class, which Zimmer said were expected and consistent with other approved JAK inhibitors.

During the call, management said there was one additional thrombosis case in the open-label extension period and no on-treatment malignancies in that period. Zimmer said the company plans to disclose the full efficacy and safety results from the extension study later.

Management Expects “Slow and Steady” Uptake

Despite physician interest and the absence of an approved targeted competitor in dermatomyositis, Roivant repeatedly characterized its expected launch trajectory as “slow and steady.” Gline said treatment practices have remained largely unchanged for decades and that changing prescribing behavior, payer processes and access patterns will take time.

Management said it has engaged with payers and expects to help patients manage prior authorizations and reimbursement requirements, but it did not provide projections for coverage levels, prescription-to-reimbursement timing or early sales. Gline said quarterly net sales would ultimately be the most useful measure of launch performance, while noting that data from orphan-drug launches can be difficult to track through third-party prescription services.

The company said it has a commercial team in place and plans to expand its field force over time. About half of dermatomyositis patients are treated at 200 specialty referral centers, according to Gline.

Further Development Plans

Roivant plans to pursue additional indications for LISRAYA. Gline said data in non-infectious uveitis are expected soon, while registrational programs in cutaneous sarcoidosis and lichen planopilaris are ongoing. He also referenced expected data from Roivant’s pulmonary hypertension program, IMVT-1402 in cutaneous lupus erythematosus, and additional rheumatoid arthritis updates before year-end.

“Dermatomyositis is just the beginning for brepocitinib,” Gline said, describing the approval as the start of a broader effort to build the LISRAYA franchise across multiple indications.

About Roivant Sciences (NASDAQ:ROIV)

Roivant Sciences is a biopharmaceutical company focused on the development and commercialization of innovative therapies through a network of subsidiary businesses known as “Vants.” Founded in 2014, Roivant acquires or in-licenses clinical-stage assets that have progressed beyond proof of concept and seeks to advance them efficiently toward regulatory approval. By organizing each program into a dedicated subsidiary, the company aims to streamline decision-making, allocate resources more effectively, and accelerate development timelines.

The core activities of Roivant involve identifying promising drug candidates across a range of therapeutic areas, including neurology, rare diseases, immunology, oncology, and women's health.

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