Evicenter
P&T meetings

Atebrioz (zilurgisertib)

Fibrodysplasia ossificans progressiva

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

Product information and disease descriptionPlace of product in therapyApproaches to treatmentCurrent treatment options and standard of care

Off-label therapies

5 evidence topics · 4 sources

Guideline position on off-label medications

“Class II Medications can be considered on off-label use with caution, at the physician’s discretion.” (opens the source at this quote in a new tab)

“There is no definitive evidence that these medications prevent or ameliorate HO after flare-ups in FOP. These medications may be considered for patients with severe, intractable FOP flare-ups.” (opens the source at this quote in a new tab)

Bisphosphonates

“There is no definitive evidence that bisphosphonates prevent or ameliorate flare-ups in FOP. However, IV bisphosphonates may be considered for the prevention of corticosteroid-associated bone loss.” (opens the source at this quote in a new tab)

“No randomized placebo-controlled trials have been conducted to date to demonstrate efficacy; one case series suggests there may be benefits for decreasing intensity of flares, but data are anecdotal.” (opens the source at this quote in a new tab)

Case-series evidence for tofacitinib and canakinumab

“In an FOP case series by Nikishina et al. 2023, levels of IL-1RA decreased in 4/5 individuals (80%) and increased in 1/5 (20%), suggesting that at least part of the mechanism of action in FOP is related to suppression of inflammatory cytokines.” (opens the source at this quote in a new tab)

“In an FOP uncontrolled case series of 4 patients by Haviv et al. 2024, flare activity was reduced by 61%-89%.” (opens the source at this quote in a new tab)

Rapamycin: case reports without disease-modifying benefit

“Several recent studies in genetic mouse models of FOP support involvement of the mechanistic target of rapamycin complex 1 (mTORC1) pathway in the pathophysiology of FOP and propose the repurposed use of rapamycin, an inhibitor of mTORC1 signaling in clinical trials for the management of FOP.” (opens the source at this quote in a new tab)

“She was treated with rapamycin (1 mg/m[2] ) for three months with a desired rapamycin trough level between 7 and 13 μg/L. Despite the treatment regimen, there was no discernible improvement.” (opens the source at this quote in a new tab)

“Following a minor rejection episode at age 10, cyclosporine was discontinued and rapamycin (sirolimus) was begun at a dose of 1–2 mg/m[2] (adjusted to therapeutic trough levels of 6–15 μg/L) for the next 18 years till the time of this report. Despite 18 years of daily rapamycin therapy, and episodic use of glucocorticoids, flare-ups and HO progressed spontaneously or following minor soft tissue trauma (Fig. 2).” (opens the source at this quote in a new tab)

“In both patients, FOP progressed despite the use of rapamycin.” (opens the source at this quote in a new tab)

Approaches with no role or that are contraindicated

“There is no evidence of efficacy with these agents. The use of these approaches is contraindicated in the management of FOP.” (opens the source at this quote in a new tab)

“The chronic use of antiangiogenic agents, calcium binders, colchicine, fluoroquinolone antibiotics, propranolol, mineralization inhibitors, PPAR-γ antagonists, and TNF-α inhibitors currently has no role in the management of FOP.” (opens the source at this quote in a new tab)

“This approach is ineffective and contraindicated in the treatment of FOP.” (opens the source at this quote in a new tab)