Mirum Pharmaceuticals Announces Positive Results from Cohort 2 of the Pivotal PROGRESS Study of ATEBRIOZ™ (zilurgisertib) in Pediatric Patients Aged 6 to <12 Years with Fibrodysplasia Ossificans Progressiva
Mirum Pharmaceuticals reported positive results from Cohort 2 of the pivotal PROGRESS study of ATEBRIOZ (zilurgisertib) in pediatric patients aged 6 to <12 years with fibrodysplasia ossificans progressiva. At Week 24, mean total HO lesion volume decreased by 8.09 cm3 on zilurgisertib versus a 9.14 cm3 increase on placebo (nominal p=0.003), with no new HO lesions in treated patients and joint mobility improvements on CAJIS. Safety was consistent with Cohort 1, which supported the September 2026 FDA approval for patients aged 12 and older. Mirum plans an sNDA submission in H1 2027 to extend the label to the 6 to <12 age group.
Key figures
- Drug Name
- ATEBRIOZ (zilurgisertib)
- Phase Of Trial
- Phase 2 (pivotal PROGRESS study)
- P value
- 0.003
- Snda submission
- H1 2027
- Cohort2 enrolled
- 25
- Cajis change placebo
- -0.09
- Treatment difference cm3
- -14
- Cajis change zilurgisertib
- -0.8
- Ho volume change placebo cm3
- 9.14
- New ho lesions zilurgisertib
- 0
- Ho volume change zilurgisertib cm3
- -8.09
AI analysis
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