US FDA approves rare bone disorder drug

18 Aug 2023 • The US FDA has approved palovarotene (Sohonos), the first treatment for fibrodysplasia ossificans progressiva (FOP), a rare and debilitating bone condition. Palovarotene is an oral drug that targets retinoic acid receptors, specifically the gamma subtype, involved in regulating skeletal development and ectopic bone formation. By interacting with these receptors and proteins within the retinoid signaling pathway, the drug reduces the formation of abnormal bone. The approval covers female patients aged 8 and older and male patients aged 10 and older with FOP. Dosage is recommended at 5 mg daily or weight-based equivalent for pediatric patients under 14 years, with potential modifications for flare-up symptoms. The drug's effectiveness was demonstrated in a phase 3 trial, reducing abnormal bone volume by 54%. Side effects include skin and mucous membrane dryness, alopecia, rash, arthralgia, and growth plate closure in children, similar to other systemic retinoid drugs. Source: Reuters | Read full story

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