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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 therapyDisease description

Diagnosis

10 evidence topics · 11 sources

Guideline: clinical diagnosis with required genetic confirmation

“The diagnosis of FOP is clinical (malformed great toes and progressive HO) but requires genetic confirmation of a pathogenic ACVR1 gene variant.” (opens the source at this quote in a new tab)

“If FOP is suspected, all elective procedures such as surgeries, biopsies, and IM immunizations should be deferred until a definitive diagnosis is confirmed.” (opens the source at this quote in a new tab)

Early clinical signs that prompt genetic testing

“Definitive genetic testing of FOP by DNA sequence analysis of the ACVR1 locus can confirm a diagnosis of FOP prior to the appearance of HO” (opens the source at this quote in a new tab)

“Clinicians should be aware of the early diagnostic signs of FOP - congenital malformation of the great toes and episodic soft tissue swelling even before the appearance of HO. This awareness should prompt genetic consultation and testing and the institution of assiduous precautions to prevent injury and iatrogenic harm.” (opens the source at this quote in a new tab)

“The clinical diagnosis of FOP can be made by simple clinical evaluation that associates rapidly appearing soft tissue lesions with malformations of the great toes.” (opens the source at this quote in a new tab)

“Individuals will often present in their first decade of life with great toe malformations and lumps/swellings on the scalp, head, neck and/or back” (opens the source at this quote in a new tab)

“FOP is the only condition with this combination of symptoms.” (opens the source at this quote in a new tab)

International survey: misdiagnosis in 87% and diagnostic delay of 4.1 years

“Incorrect diagnoses were given initially to 87% of individuals with FOP.” (opens the source at this quote in a new tab)

“The most common incorrect diagnosis was cancer (32%). The mean period from the onset of symptoms to correct diagnosis was 4.1 years, and the median number of physicians consulted before the correct diagnosis of FOP was 6. For 67% of patients, unnecessary invasive procedures (biopsies) were performed; 68% received inappropriate therapies. Forty-nine percent of all patients reported permanent loss of mobility resulting from invasive medical interventions that caused posttraumatic ossification.” (opens the source at this quote in a new tab)

FOP Registry: time to diagnosis and number of providers consulted

“Data from the FOP Registry (as of December 2020) show that on average, individuals with FOP receive a correct diagnosis around 1.5 years following symptom onset, with over 50% of individuals initially misdiagnosed” (opens the source at this quote in a new tab)

“According to recent FOP Registry data, individuals with FOP receive a correct diagnosis after seeing an average of 3.3 HCPs” (opens the source at this quote in a new tab)

“According to the FOP Registry, 53.5% of patients had received a misdiagnosis prior to correct diagnosis.” (opens the source at this quote in a new tab)

Initial evaluation and diagnosing provider in the IFOPA registry

“Most patients consulted a pediatrician (27.4%) or an orthopaedic physician (12%) for their initial diagnostic evaluation, and most often received their correct diagnosis from a geneticist (18.1%) [data not shown].” (opens the source at this quote in a new tab)

China: misdiagnosis in 78.8% and iatrogenic harm in 62.7%

“Up to 78.8% of patients would have been misdiagnosed at least once, with tumors (40.9%) being the most common misdiagnosed condition. 62.7% of patients received iatrogenic harm, with surgeries (26.9%), biopsies (25.4%), and punctures (17.9%) being the most common iatrogenic injuries experienced.” (opens the source at this quote in a new tab)

“Only 23.9% of patients received a correct diagnosis at their initial hospital visit (Table S1).” (opens the source at this quote in a new tab)

Age at diagnosis

“Mean age at FOP diagnosis was 6.9 years (range: birth to 63 years); the median age was 5 years.” (opens the source at this quote in a new tab)

“Mean age at diagnosis was 10 years, and above 18 years for 14.9% of the patients.” (opens the source at this quote in a new tab)

“Based on the age group at index date, the mean (SD) age at diagnosis was 2.43 (2.34) yr for individuals aged <10 yr at index date, 4.64 (4.59) years for individuals aged 10-20 yr, 5.91 (4.90) years for individuals aged 20-30 yr, and 9.29 (8.29) yr for individuals aged ≥30 yr (p = .0189 for the difference between age at diagnosis for age groups).” (opens the source at this quote in a new tab)

Age at symptom onset and age at diagnosis in the IFOPA registry
MeasureAge at symptom onset (years)Age at diagnosis (years)
n“209”“208”
Mean (SD)“5.3 (6.13)”“7.2 (7.18)”
Median“3.0”“5.0”
Minimum, maximum“0.1, 45”“0.1, 48.0”
Pediatric cohort: biopsy before referral and scalp nodules as an early sign

“Four patients underwent biopsy prior to referral to our institution. One was coincidentally associated with Ewing sarcoma.” (opens the source at this quote in a new tab)

“Scalp nodules could be a transient finding with spontaneous regression and were an early manifestation in 2.” (opens the source at this quote in a new tab)

Newborn screening

“Therefore, the routine screening of newborns to diagnose FOP, either clinically or genetically, is unlikely to become more widespread in the near future.” (opens the source at this quote in a new tab)