What's Happening?
A phase 2 trial investigating the efficacy of palovarotene for patients with multiple hereditary exostosis (MHE) was terminated early, resulting in an underpowered study. Conducted between March 2018 and December 2019, the trial involved 194 patients,
with 193 receiving at least one dose of palovarotene or placebo. The study aimed to assess the drug's ability to prevent new osteochondromas (OCs), but due to early termination, no patients completed the planned treatment schedule. Efficacy imaging at 12 months showed no statistically significant differences in the annualized rate of new OCs between treatment groups. Safety data indicated that treatment-emergent adverse events (TEAEs) were more frequent in palovarotene-treated patients, with common TEAEs including mucocutaneous issues. Serious TEAEs were reported in a small number of patients, with no deaths or withdrawals due to adverse events.
Why It's Important?
The early termination of the palovarotene trial highlights challenges in developing effective treatments for MHE, a condition characterized by the growth of multiple benign bone tumors. The lack of significant efficacy in preventing new OCs suggests that palovarotene may not be a viable treatment option for MHE, impacting patients and healthcare providers seeking effective therapies. The trial's safety findings also underscore the need for careful consideration of adverse effects in drug development. The results may influence future research directions and regulatory decisions regarding palovarotene and similar treatments.
What's Next?
Further research is needed to explore alternative treatments for MHE, as the current trial's results do not support the continued use of palovarotene for this condition. Researchers may focus on identifying new therapeutic targets or optimizing existing treatments to improve efficacy and safety. Regulatory agencies will likely review the trial data to determine the future of palovarotene in clinical use. Patients and healthcare providers will need to consider other management strategies for MHE in the absence of effective pharmacological options.
