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Atebrioz (zilurgisertib)

Fibrodysplasia ossificans progressiva

Also known as INCB000928
Regulatory submission
NDA submitted by May 2026
Last updated
96 sources

Economic information and modeling reportBudget impact model

Epidemiology and eligible population inputs

7 evidence topics · 8 sources

Zilurgisertib: U.S. and worldwide patient population stated by the manufacturer

“Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, progressive genetic disease affecting approximately 300 people in the United States and 900 worldwide.” (opens the source at this quote in a new tab)

Zilurgisertib: manufacturer estimate of current treatment exposure

“we actually estimate there were more [FOP] patients on clinical studies than on commercial drugs. Which we take as a real statement” (opens the source at this quote in a new tab)

Palovarotene: sales and patient uptake reported by the manufacturer

“Sohonos: flat sales impacted by limited number of new patients in the U.S.” (opens the source at this quote in a new tab)

Sohonos sales, millions of eurosFY 2025FY 2024
Total“20.7”“20.8”
North America“11.6”“14.1”
Zilurgisertib: analyst statements on the U.S. patient population and expected treatment preference

“Leerink Partners estimates there are about 300 FOP patients in the U.S. Analyst Joseph Schwartz said in a research note that diagnosis rates for the disorder could rise as new therapies create awareness of the disease and genetic testing increases. He added that clinicians have told Leerink that that they expect patients will prefer Atebrioz’s daily oral formulation versus monthly infusions of Regeneron’s drug.” (opens the source at this quote in a new tab)

“Leerink estimates the drug could achieve $200 million in peak sales, though that figure could change when Mirum releases pricing information.” (opens the source at this quote in a new tab)

United States: age distribution of 284 people with FOP

“Of 284 patients with age data, the average age was 29.3 years; 31 (11%) were 0 to < 10 years, 65 (23%) were ≥ 10 to < 20 years, 63 (22%) were ≥ 20 to < 30 years, 58 (20%) were ≥ 30 to < 40 years, and 67 (24%) were ≥ 40 years.” (opens the source at this quote in a new tab)

Palovarotene budget impact analysis submitted to CADTH: population and uptake inputs
ParameterSponsor’s estimate (year 1 / year 2 / year 3)
Prevalence of FOP“0.000056%”
Proportion of FOP patients aged ≥ 8 years (female) or ≥ 10 years (male)“88%”
Proportion of patients diagnosed“90%”
Proportion of diagnosed patients receiving treatment“100%”
Proportion of patients with public coverage“Jurisdiction-specific”
Number of patients eligible for drug under review“10 / 10 / 10”
Uptake of palovarotene plus SoC, new drug scenario“35% / 40% / 45%”
Palovarotene budget impact analysis: CADTH appraisal of the population inputs

“The sponsor assumed that 90% of patients with FOP would be correctly diagnosed owing to difficulty in correctly diagnosing FOP.” (opens the source at this quote in a new tab)

“For year 1, assuming 35% uptake results in 3 patients anticipated to receive palovarotene plus SoC.” (opens the source at this quote in a new tab)

“In the CADTH base case, CADTH increased the uptake of palovarotene such that 5 patients would receive palovarotene plus SoC in year 1.” (opens the source at this quote in a new tab)

“CADTH’s estimated budget impact of reimbursing palovarotene is based on an estimated prevalence of 0.56 per million, based on Canadian registry data from 2016, resulting in approximately 19 patients with FOP in Canada; however, the Canadian FOP Network estimates that there are 34 patients with FOP in Canada.” (opens the source at this quote in a new tab)