For years, there have been few – or no – therapeutic options in Canada for those with fibrodysplasia ossificans progressiva (FOP), a rare genetic connective tissue disorder. However, this has recently changed. In a January 24 news release, biopharmaceutical company Ipsen shared that Sohonos (palovarotene capsules), a therapy acquired by Ipsen in April 2019, was now approved for use by Health Canada. In particular, the treatment is now approved for use in female children aged 8+, male children aged 10+, and adults.
About Fibrodysplasia Ossificans Progressiva (FOP)
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder resulting from ACVR1 gene mutations. The condition is characterized by the formation of bone outside of the skeletal structures, such as in tendons, muscle, or ligaments. Ossification usually begins around the neck or shoulders, but later progresses to the limbs. However, one sign may be observed at birth: big toe or thumb malformations. FOP is either inherited in an autosomal dominant pattern (requiring only one defective gene) or acquired spontaneously. An estimated 1 in every 1.6 million people is born with FOP. Symptoms include:
- Progressive loss of mobility
- Restricted movement
- Difficulty speaking, eating, or breathing
- Muscle pain and inflammation
- Toe and finger malformations
- Chronic inflammation
- Scoliosis or kyphosis
- Entrapment neuropathies
Sohonos (Palovarotene Capsules)
Sohonos is an orally administered retinoic-acid receptor gamma (RARγ) agonist. FOP is characterized by heterotopic ossification, or the process of tissue transformation into bone. Sometimes, this ossification may occur after a “flare,” in which patients experience painful soft tissue inflammation. Ultimately, Sohonos is designed to both stop these flares and heterotopic ossification. If you would like to learn more about palovarotene overall, take a look at this helpful guide from the International Fibrodysplasia Ossificans Progressiva Association (IFOPA).
The approval by Health Canada follows data from the Phase 3 MOVE clinical trial, which is evaluating the therapy’s safety, efficacy, and tolerability.
