US FDA approves Sohonos for FOP fibrodysplasia ossificans progressiva

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FOP is a very rare genetic connective tissue disorder characterized by the abnormal development of bone in areas of the body where bone is not normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. Specifically, this disorder causes the body’s skeletal muscles and soft connective tissues to undergo a metamorphosis, essentially a transformation into bone, progressively locking joints in place and making movement difficult or impossible.

FOP is caused by the mutation of a gene (ACVR1) in the bone morphogenetic protein (BMP) pathway, which is important during the formation of the skeleton in the embryo and the repair of the skeleton following birth.

FOP impacts the lives of an estimated 900 people globally.

By the age of 30 years old, the majority of people with FOP require a wheelchair and full-time caregiver assistance.

Ipsen Pharma’s Sohonos the first and only oral treatment for FOP.

Sohonos is an oral medicine with particular selectivity for the gamma subtype of retinoic-acid receptors. RAR are an important regulator of skeletal development and ectopic bone in the retinoid signaling pathway.

The medicine is designed to mediate the interactions between the receptors, growth factors and proteins within the retinoid signaling pathway to reduce new abnormal bone formation in FOP.

The recommended dosing for Sohonos includes a chronic daily dosage of 5 mg.

Sohonos received Orphan Drug and Breakthrough Therapy Designations.

 

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