Sohonos
Evidence Grade A — Regulatory approved. 60 published studies. 12 registered clinical trials.
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Palovarotene (sold as Sohonos) is an oral medication for fibrodysplasia ossificans progressiva (FOP) — an extraordinarily rare genetic condition (approximately 1 in 1.3 million people) where the body's soft tissues progressively turn into bone, gradually encasing the skeleton in a second layer of bone that locks joints in place. It is the first and only treatment for FOP. Palovarotene is not a peptide.
60 published studies: 40 human, 9 animal, 8 in-vitro, 18 reviews
Palovarotene (Sohonos) is approved for FOP. The Phase III MOVE trial (107 patients — representing approximately 12% of the global FOP population) initially did not meet statistical significance on its pre-specified primary analysis. However, a post-hoc re-analysis with corrected placebo data showed a 54% reduction in new heterotopic ossification volume.
The approval pathway was complex given the ultra-rare nature of FOP. Palovarotene is not a peptide. Important safety considerations include premature growth plate closure in growing children, requiring monitoring. FOP has no other approved treatment.
Palovarotene selectively activates the retinoic acid receptor gamma, which blocks the aberrant bone-forming signalling pathway caused by the genetic mutation in FOP. By inhibiting this pathway, palovarotene reduces the abnormal bone formation that progressively restricts movement in FOP patients. The drug does not reverse existing heterotopic bone but aims to prevent new bone formation episodes.
FOP is so rare that fewer than 1,000 patients exist worldwide, making traditional clinical trials exceptionally challenging. The Phase III MOVE trial (107 patients — representing about 12% of the entire global FOP population) initially did not meet statistical significance on its planned primary analysis, but a corrected re-analysis showed a 54% reduction in new abnormal bone formation. The approval pathway has been uneven: the FDA approved it in August 2023 and Health Canada in January 2022, but the European Medicines Agency refused marketing authorisation in July 2023, citing insufficient evidence. Important safety concerns include premature growth plate closure in growing children (which could affect normal bone growth) and risk to unborn babies during pregnancy — both reflected in boxed warnings. As there is no other treatment for FOP, the risk-benefit calculation is different from conditions with existing alternatives.
A Study of the Blood Levels of Palovarotene in Participants With Abnormal Liver Function Compared to Healthy Adult Participants After Intake of a Single Dose
A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.
Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult Subjects
Study to Assess Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of Palovarotene Ophthalmic Solution in Healthy Adult Subjects
Health Canada Market Authorisation
FDA ORIG 1
FDA SUPPL 2
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