Name: Atebrioz (zilurgisertib) in Fibrodysplasia ossificans progressiva: Heterogeneity of treatment effect
Published: 2026-09-27T12:28:00-04:00

Product information and disease description Place of product in therapy Approaches to treatment

Heterogeneity of treatment effect

5 evidence topics · 4 sources

Variability of disease course among individuals

“However, there is great variability among individuals in terms of course and severity.” (opens the source at this quote in a new tab)
“Although there are common physical features shared by every person who has FOP, there are differences among individuals that may alter the potential benefits or risks of any medication or class of medications discussed here.” (opens the source at this quote in a new tab)

ACVR1 variant and eligibility in the pivotal trial

“An estimated 97% of individuals with FOP have this recurrent variant (ACVR1 R206H). Approximately 3% of affected individuals have different pathogenic variants in ACVR1” (opens the source at this quote in a new tab)

“Inclusion criteria included clinical diagnosis of FOP, patient-reported FOP disease activity within 1 year of the screening visit (i.e., FOP flare-ups, worsening joint function, or radiographic progression of HO), and a baseline Cumulative Analogue Joint Involvement Scale (CAJIS) score ≤ 23.” (opens the prescribing information at this quote)

Age and disease severity of the enrolled population

“The median age of enrolled patients was 16 years (range: 12 to 64 years), 56% were 12 to 16 years of age” (opens the prescribing information at this quote)
“All patients had a baseline CAJIS score of ≤ 23 and 84% of patients had a score < 18 (range: 2-22).” (opens the prescribing information at this quote)
“Clinical studies of ATEBRIOZ did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients.” (opens the prescribing information at this quote)

Safety and exposure by age group

“Safety was similar between adults and adolescents; this was also observed during the OLE” (opens the source at this quote in a new tab)

“In pediatric patients with FOP (12 to 16 years of age), the estimated steady-state AUC and Cmax for zilurgisertib was 11478 nM·h and 883 nM, respectively.” (opens the prescribing information at this quote)

Age-dependent risk with the retinoic acid receptor gamma agonist

“In MOVE, the 80 patients aged <18 yr (age range: 4-17 yr) received palovarotene for a mean (SD) of 166.0 (75.2) wk. Of these, 24/80 (30.0%) had clinical events consistent with PPC, all of which occurred in those aged <14 yr” (opens the source at this quote in a new tab)