WILMINGTON, DE — Incyte (Nasdaq: INCY) won U.S. approval for zilurgisertib, a treatment it developed for fibrodysplasia ossificans progressiva, giving the company a regulatory milestone and a transferable priority-review voucher while partner Mirum Pharmaceuticals prepares to commercialize the drug.
The Food and Drug Administration approved the therapy, branded Atebrioz, for patients age 12 and older with fibrodysplasia ossificans progressiva, or FOP, to reduce the volume of new heterotopic ossification.
Incyte licensed worldwide development and commercialization rights for the drug to Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM). Mirum expects to make Atebrioz commercially available in the U.S. in October.
The FDA also awarded Incyte a Rare Pediatric Disease Priority Review Voucher as part of the approval. Such vouchers can be used to accelerate review of a future drug application that otherwise would not qualify for priority review.
Atebrioz is a once-daily oral inhibitor of activin receptor-like kinase 2, or ALK2. In FOP, pathogenic variants in the ACVR1 gene abnormally activate ALK2, contributing to bone formation in muscles, tendons, ligaments and other soft tissues.
The disease can progressively restrict movement and cause significant disability as heterotopic ossification accumulates.
Approval was based on Cohort 1 of the Phase III PROGRESS study in adult and pediatric patients age 12 and older.
At 24 weeks, patients receiving zilurgisertib had a mean 3.2-cubic-centimeter decrease in total new heterotopic ossification lesion volume, compared with a 24.6-cubic-centimeter increase among patients receiving placebo. Mirum and Incyte said the treatment effect was maintained through 48 weeks of an open-label extension.
The most commonly reported adverse reactions during the placebo-controlled portion of the study were headache, joint pain, upper respiratory tract infection, nosebleed and nausea. The companies said most adverse events were mild or moderate, with none leading to treatment discontinuation or dose reduction.
“Having another treatment option is meaningful in a progressive disease like FOP, particularly for adolescents who may be earlier in the course of their disease,” Robert Pignolo, lead investigator for the PROGRESS study and a professor at the Mayo Clinic College of Medicine, said.
The recommended Atebrioz dose is 100 milligrams orally once daily.
Mirum plans to distribute the drug through its Mirum Access Plus patient-support program, which provides insurance and access assistance and financial support for eligible patients. The company said qualifying patients may pay as little as $0 per month.
European regulators are also reviewing zilurgisertib based on data from PROGRESS Cohort 1.
Development is continuing in younger children. Enrollment has been completed in a cohort of patients ages 6 to under 12, while enrollment is underway in another cohort evaluating children ages 2 to under 12.
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