Name: Atebrioz (zilurgisertib) in Fibrodysplasia ossificans progressiva: Activin A monoclonal antibodies
Published: 2026-09-27T12:28:00-04:00

Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Activin A monoclonal antibodies

4 evidence topics · 5 sources

Phase 3 OPTIMA trial: new lesions and flare-ups

“There were a total of 19 new HO lesions observed in 5/21 (24%) patients receiving placebo compared to 2 total lesions in 2/23 (9%) patients or 1 lesion in 1/19 (5%) patients treated with PASATRU 10 mg/kg or 3 mg/kg every 4 weeks, respectively, at Week 56 (see Table 2).” (opens the source at this quote in a new tab)
“The number of clinician-assessed flare-ups through Week 56 was 9, 53, and 66 for PASATRU 10 mg/kg, 3 mg/kg, and placebo, respectively.” (opens the source at this quote in a new tab)
“Changes in the proportion of patients with patient-reported flare-ups through Week 56 were not significantly different between placebo and PASATRU treatment groups.” (opens the source at this quote in a new tab)

Warnings for infection and epistaxis

“Skin and soft tissue infections requiring treatment and/or hospitalization, including abscesses and cellulitis [see Adverse Reactions (6.1)], have occurred in patients receiving PASATRU in clinical trials.” (opens the source at this quote in a new tab)
“In clinical trials in the FOP population, one serious case of spontaneous epistaxis occurred that required hospitalization for nasal packing and application of a clotting agent.” (opens the source at this quote in a new tab)

Phase 2 LUMINA-1 trial

“The primary efficacy end point in period 1 (total lesion activity by PET–CT) was not met ( P = 0.0741). As the development of new HO lesions was suppressed in period 1, the primary efficacy end point in period 2 was prospectively changed to the number of new HO lesions versus period 1. No placebo patients crossing over to garetosmab developed new HO lesions (0% in period 2 versus 40.9% in period 1; P = 0.0027).” (opens the source at this quote in a new tab)
“Five deaths (5 of 44; 11.4%) occurred in the open-label period and, while considered unlikely to be related, causality cannot be ruled out.” (opens the source at this quote in a new tab)

Guideline classification before approval

“Blocks Activin A signaling through mutant ACVR1/ALK2” (opens the source at this quote in a new tab)
“Not applicable at Present time; Ongoing Phase III clinical trial” (opens the source at this quote in a new tab)