Drug intelligence / Profile preview

zilurgisertib

Development stage
Approved
Lead developer
Mirum Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

**Zilurgisertib** is an orally administered, selective, ATP-competitive small-molecule inhibitor of activin receptor-like kinase 2, also called activin receptor type-1. It was originated by Incyte and licensed worldwide to Mirum for development and commercialization. In the United States, it is marketed as **Atebrioz** and is approved to reduce the volume of total new heterotopic ossification in adults and pediatric patients aged 12 years and older with fibrodysplasia ossificans progressiva. By inhibiting pathogenic ALK2 signaling driven by ACVR1 variants, zilurgisertib suppresses aberrant bone morphogenetic protein pathway activity associated with heterotopic bone formation. It has also been studied for anemia associated with myelofibrosis, myelodysplastic syndromes, and multiple myeloma. ([cancer.gov](https://www.cancer.gov/publications/dictionaries/cancer-drug/def/zilurgisertib))

Brand names
Atebrioz
Other names
zilurgisertib
02

Targets

ACVR1 (Activin receptor type I)

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