Though not without a bump along the way, Roivant-backed Priovant Therapeutics has pushed its dual TYK2/JAK1 inhibitor across the FDA finish line, securing its first approval in a rare skin disease.
The FDA has given the green light to Priovant’s brepocitinib, which will now be marketed under the commercial moniker Lisraya as a once-a-day pill for adults with dermatomyositis (DM), an autoimmune condition marked by progressive muscle weakness and extensive, painful and itchy skin lesions.
Lisraya now holds the distinction of being the first targeted therapy approved in DM, which is traditionally treated with chronic high-dose steroids. The disease can take a toll on patients’ quality of life by way of physical disability, skin and muscle pain, cutaneous disfigurement and sensitivity to light and touch, Priovant said in an Aug. 27 release.
The drug boasts a broad label, too, cleared for use in adult DM patients without restriction based on level of disease activity, clinical presentation or previous treatment, Priovant said. The drug can be used as both an alternative therapy or an add-on to current non-targeted DM medicines, the company explained.
The approval was propped up by data from Priovant’s late-stage Valor trial, which the company touts as the biggest DM study ever conducted.
After a year of dosing in the trial—which assessed two doses of Lisraya against placebo in 241 people with DM—Priovant observed a mean myositis Total Improvement Score (TIS) of 46.5 on the high dose, versus 31.2 on placebo, allowing the study to meet its primary endpoint.
In its approval announcement, Priovant noted that improvements to TIS in patients on Lisraya came as early as four weeks, increased over time and were sustained through the end of the trial period. Additionally, 55% of patients treated with Lisraya were able to achieve both moderate or better improvement on the TIS alongside minimal or no steroid use by the study’s end, as compared to 30% of patients in the control arm.
In attempting to frame Lisraya’s potential merit in the DM community, Priovant highlighted patients’ own impression of their experience, noting that when asked about the overall activity of their disease, patients on Lisraya reported more than four times as much improvement as those who got the dummy drug.
The company also pointed to Lisraya’s potential to help restore a degree of independence to DM patients’ lives.
As part of the JAK family, Lisraya carries an expected box warning for serious infections, death, malignancy, major adverse cardiovascular events and thrombosis. The more common side effects logged in the Valor study included upper respiratory tract infection, headache, fatigue, urinary tract infection, nausea and flu, among other reactions.
In an Aug. 27 statement, Priovant’s CEO, Ben Zimmer called Lisraya’s approval “a historic moment for the dermatomyositis community.”
“I am thrilled that adults with dermatomyositis finally have a fundamentally new treatment option— one specifically designed to target the biology of their disease and shown to meaningfully improve how patients feel and function in their daily lives,” he said.
The green light marks the first for Priovant, which was formed by Roivant in 2022 with a Pfizer deal for brepocitinib and the TYK2 inhibitor ropsacitinib.
While the company had settled on dermatomyositis as the drug’s lead indication in the pivot into phase 3, a phase 2 lupus trial flop in 2023 put a blemish on the drug’s clinical track record.
Priovant is eventually hoping to score additional indications for the molecule, which is also in phase 3 testing in noninfectious uveitis and recently began enrolling patients in a late-stage study in cutaneous sarcoidosis.