Palovarotene
Palovarotene is an orally administered retinoic acid receptor agonist specifically developed for the treatment of Fibrodysplasia Ossificans Progressiva (FOP), a rare genetic disorder characterized by progressive heterotopic ossification where bone forms abnormally in muscles, tendons, and ligaments. By targeting the gain-of-function mutation in the ACVR1/ALK2 receptor, palovarotene helps reduce the formation of new bone lesions in FOP patients. This medication represents a specialized treatment approach for a condition that was historically managed only with supportive care.
Palovarotene is a retinoic acid receptor agonist used to reduce heterotopic ossification in patients with Fibrodysplasia Ossificans Progressiva (FOP), a rare genetic disorder causing progressive bone formation in soft tissues.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- M09AX11
- RxNorm
- 2663954
- Generic
- Palovarotene
- Class
- atc-m09
- Brands
- Sohonos
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Palovarotene is indicated for the treatment of Fibrodysplasia Ossificans Progressiva (FOP) in adults and children aged 8 years and older for females and 10 years and older for males. FOP is a rare, progressive genetic disorder marked by abnormal bone formation in soft tissues, leading to severe physical disability.
Palovarotene is an orally bioavailable retinoic acid receptor (RAR) agonist. In FOP patients, a gain-of-function mutation in the BMP type I receptor ALK2 (ACVR1) drives abnormal bone formation. Palovarotene works by modulating the RAR signaling pathway, which inhibits the chondrocyte and osteoblast differentiation responsible for heterotopic ossification formation.
Palovarotene should be initiated and supervised by physicians experienced in managing FOP. Common adverse effects include dry skin, lip abnormalities, and musculoskeletal pain. In pediatric patients, premature epiphyseal plate closure and growth retardation have been reported. Regular monitoring of growth and skeletal development is recommended in children and adolescents during treatment.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy