Palovarotene in fibrodysplasia ossificans progressiva: review and perspective.
Publication/Presentation Date
2-1-2025
Abstract
INTRODUCTION: Palovarotene is a retinoic acid receptor gamma agonist that was studied in phase-2 and phase-3 clinical trials for the inhibition of new heterotopic ossification (HO) in fibrodysplasia ossificans progressiva (FOP). Despite numerous setbacks and regulatory delays, palovarotene is now the first approved FOP treatment in the U.S.A., Canada and Australia but remains unapproved in Europe where concerns surrounding the drug and its path to regional market authorization persist.
AREAS COVERED: The developmental history of palovarotene and an overview of the clinical trials and the regulatory approval journey are discussed by global FOP experts.
EXPERT OPINION: While post hoc analyses indicate that palovarotene may have modest benefits for the inhibition of new HO formation in FOP, a number of limitations and concerns remain about its generalized use. Although the long-term risks and benefits of treatment with palovarotene remain unknown, the regional approval of palovarotene marks a milestone for the FOP community at the very beginning of a new era of clinical trials.
Volume
26
Issue
3
First Page
291
Last Page
299
ISSN
1744-7666
Published In/Presented At
Verheij, V. A., Diecidue, R. J., Botman, E., Harrington, J. D., Haga, N., Hidalgo-Bravo, A., Delai, P. L. R., Madhuri, V., Al Mukaddam, M., Zhang, K., Cho, T. J., Morhart, R., Keen, R., De Cunto, C. L., Friedman, C. S., Grunwald, Z., Zasloff, M., Netelenbos, J. C., Hsiao, E., Kaplan, F. S., … Eekhoff, E. M. W. (2025). Palovarotene in fibrodysplasia ossificans progressiva: review and perspective. Expert opinion on pharmacotherapy, 26(3), 291–299. https://doi.org/10.1080/14656566.2025.2452938
Disciplines
Business Administration, Management, and Operations | Health and Medical Administration | Management Sciences and Quantitative Methods
PubMedID
39834193
Department(s)
Administration and Leadership
Document Type
Article