Clementia is charting a new course to develop effective new treatments for people who have none.

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About FOP

Fibrodysplasia ossificans progressiva, or FOP, is a rare, severely disabling congenital myopathy characterized by heterotopic ossification (HO), or bone that forms outside the normal skeleton. This extra-skeletal bone progressively restricts movement by locking joints and eventually imprisons affected persons in a “second skeleton.” FOP is caused by a mutation in the ACVR1 gene, resulting in excess BMP signaling in the bone morphogenetic (BMP) pathway, a key pathway controlling bone growth and development. Nearly 100 percent of newborns with FOP have a hallmark toe malformation, with both big toes shortened and bent inwards.

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About Palovarotene

Palovarotene is being investigated as a treatment for patients with debilitating bone and other diseases with high unmet medical need. Preliminary Phase 2 data in subjects with FOP mirrors the decrease in HO volume observed in lab studies in mouse models of FOP, and support the initiation of a confirmatory Phase 3 program. Palovarotene also inhibits the formation of osteochondromas (OCs) in mouse models of MO, supporting development in this indication.

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