Fibrodysplasia Ossificans Progressiva Medication

Updated: Aug 18, 2023
  • Author: Robert A Schwartz, MD, MPH; Chief Editor: Dirk M Elston, MD  more...
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Medication Summary

Palovarotene was the first drug approved for fibrodysplasia ossificans progressiva (FOP). [38]  Clinical trials have been underway with fidresertib, saractinib, garetosmab (REGN 2477), and rapamycin. [47, 48]


Retinoic Acid Receptor Gamma Agonists

Class Summary

In patients with fibrodysplasia ossificans progressiva, abnormal bone formation, including heterotrophic ossification, is driven by a gain-of-function mutation in the bone morphogenetic protein (BMP) type I receptor ALK2 (ACVR1). By binding to RARγ, palovarotene decreases the BMP/ALK2 downstream signaling pathway by inhibiting phosphorylation of SMAD1/5/8, which reduces ALK2/SMAD-dependent chondrogenesis and osteocyte differentiation resulting in reduced endochondral bone formation. 

Palovarotene (Sohonos)

Indicated for reduction in volume of new heterotopic ossification in adults and pediatric patients aged ≥8 years for females and ≥10 years for males with fibrodysplasia ossificans progressiva (FOP).