The US Food and Drug Administration approved zilurgisertib (Atebrioz) to reduce total new heterotopic ossification volume in adult and pediatric patients aged 12 years and older with fibrodysplasia ossificans progressiva, according to an FDA press release .

Fibrodysplasia ossificans progressiva is a rare genetic disease resulting from a mutation in activin A receptor-type 1, which is involved in controlling new bone growth. Muscle, tendons, ligaments, and other connective tissues progressively become bone in patients with the disease, resulting in restricted movement, deformities, severe disability, and early mortality.

Zilurgisertib was evaluated in a randomized, double-blind, placebo-controlled trial that included 63 patients with fibrodysplasia ossificans progressiva. For the first 24 weeks, patients were randomly assigned to zilurgisertib 100 mg once daily or placebo. Patients subsequently received zilurgisertib 100 mg daily during a 292-week, single-arm, open-label extension.

The efficacy assessment compared changes from baseline in total new heterotopic ossification volume between the treatment and placebo groups during the double-blind period. Whole-body computed tomography scans were used to measure total new heterotopic ossification volume.

Mean total new heterotopic ossification volume declined by 3.2 cm³ in the zilurgisertib group and increased by 24.6 cm³ in the placebo group at week 24.

Zilurgisertib is administered orally at a recommended starting dosage of 100 mg once daily and may be taken with or without food. Most common adverse effects include headache, joint pain, upper respiratory tract infection, nosebleeds, and nausea. The prescribing information identifies certain medications that should not be used with zilurgisertib.

Animal data indicate that zilurgisertib can cause fetal harm. Effective contraception is recommended for patients of reproductive potential. If pregnancy occurs, treatment should be discontinued immediately and the patient should contact their health care provider.

For this indication, the FDA granted zilurgisertib fast track, priority review, and orphan drug designations.

Source: US Food and Drug Administration