Document Type
Article
Publication 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.
Recommended Citation
Verheij, Vincent; Diecidue, Robert; Botman, Esmée; Harrington, Joseph; Haga, Nobuhiko; Hidalgo-Bravo, Alberto; Delai, Patricia; Madhuri, Vrisha; Al Mukaddam, Mona; Zhang, Keqin; Cho, Tae-Joon; Morhart, Rolf; Keen, Richard; De Cunto, Carmen; Friedman, Clive; Grunwald, Zvi; Zasloff, Michael; Netelenbos, J. Coen; Hsiao, Edward; Kaplan, Frederick; Pignolo, Robert; Scott, Christiaan; and Eekhoff, Elisabeth Marelise, "Palovarotene in Fibrodysplasia Ossificans Progressiva: Review and Perspective" (2025). Department of Oral and Maxillofacial Surgery Faculty Papers. Paper 5.
https://jdc.jefferson.edu/omsfp/5
Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.
Language
English
Included in
Heterocyclic Compounds Commons, Investigative Techniques Commons, Musculoskeletal Diseases Commons, Organic Chemicals Commons, Pathological Conditions, Signs and Symptoms Commons
Comments
This article is the author's final published version in Expert opinion on pharmacotherapy, Volume 26, Issue 3, 2025, Pages 291 - 299.
The published version is available at https://doi.org/10.1080/14656566.2025.2452938.
Copyright © 2025 The Author(s)