TY - JOUR
T1 - Palovarotene in fibrodysplasia ossificans progressiva
T2 - review and perspective
AU - Verheij, Vincent A.
AU - Diecidue, Robert J.
AU - Botman, Esmée
AU - Harrington, Joseph D.
AU - Haga, Nobuhiko
AU - Hidalgo-Bravo, Alberto
AU - Delai, Patricia L. R.
AU - Madhuri, Vrisha
AU - Al Mukaddam, Mona
AU - Zhang, Keqin
AU - Cho, Tae-Joon
AU - Morhart, Rolf
AU - Keen, Richard
AU - de Cunto, Carmen L.
AU - Friedman, Clive S.
AU - Grunwald, Zvi
AU - Zasloff, Michael
AU - Netelenbos, J. Coen
AU - Hsiao, Edward
AU - Kaplan, Frederick S.
AU - Pignolo, Robert J.
AU - Scott, Christiaan
AU - Eekhoff, Elisabeth Marelise W.
N1 - Publisher Copyright:
© 2025 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2025
Y1 - 2025
N2 - 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.
AB - 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.
KW - Palovarotene
KW - fibrodysplasia ossificans progressive
KW - heterotopic ossification
KW - outcome treatment trials
KW - retinoic acid receptor gamma agonist
KW - review
UR - https://www.scopus.com/pages/publications/85217405225
U2 - 10.1080/14656566.2025.2452938
DO - 10.1080/14656566.2025.2452938
M3 - Review article
C2 - 39834193
SN - 1465-6566
VL - 26
SP - 291
EP - 299
JO - Expert opinion on pharmacotherapy
JF - Expert opinion on pharmacotherapy
IS - 3
ER -