The US Food and Drug Administration (FDA) has approved Atebrioz (zilurgisertib) tablets to reduce the volume of total new heterotopic ossification (HO) in patients ages 12 years and older with fibrodysplasia ossificans progressiva (FOP).
FOP is a disorder in which skeletal muscle and connective tissue are gradually ossified. This condition leads to bone formation outside the skeleton that restricts movement. This process generally becomes noticeable in early childhood, starting with the neck and shoulders and moving down the body and into the limbs. People with FOP are born with abnormal big toes which can be helpful in making the diagnosis. Trauma, such as a fall or invasive medical procedure, or a viral illness may trigger episodes of muscle swelling and inflammation. These flare ups last for several days to months and often result in permanent bone growth in the injured area. FOP is almost always caused by a genetic mutation in the ACVR1 gene.
Atebrioz is a once-daily oral activin receptor-like kinase 2 (ALK2) inhibitor designed to reduce the volume of total new HO in patients ages 12 years and older with FOP.
The approval was based on data from Cohort 1 of the PROGRESS clinical trial, a global, randomized, double-blind, placebo-controlled phase 2 study. At week 24, mean total new HO lesion volume decreased by 3.2 cm3 in patients receiving zilurgisertib compared with an increase of 24.6 cm3 in patients receiving placebo. Treatment effects were maintained through week 48 of the open-label extension.
Additionally, zilurgisertib was generally well tolerated during the 24-week placebo-controlled period of the study. The most common adverse reactions were headache, arthralgia, upper respiratory tract infection, epistaxis and nausea. Most adverse events were mild or moderate in severity, and no adverse events led to treatment discontinuation or dose reduction.
Alongside the approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Incyte. The voucher can be used for a subsequent drug application that would not otherwise qualify for a priority review.
For more information, visit https://www.biospace.com/press-releases/mirum-pharmaceuticals-and-incyte-announce-u-s-fda-approval-of-atebrioz-zilurgisertib-for-adult-and-pediatric-patients-with-fibrodysplasia-ossificans-progressiva
To learn more about rare musculoskeletal diseases, visit https://checkrare.com/diseases/musculoskeletal-diseases/