Palovarotene in fibrodysplasia ossificans progressiva: review and perspective

被引：1

作者：

Verheij, Vincent A. ^{[1
]}

Diecidue, Robert J. ^{[2
]}

Botman, Esmee ^{[1
]}

Harrington, Joseph D. ^{[2
]}

Haga, Nobuhiko ^{[3
]}

Hidalgo-Bravo, Alberto ^{[4
]}

Delai, Patricia L. R. ^{[5
]}

Madhuri, Vrisha ^{[6
]}

Al Mukaddam, Mona ^{[7
]}

Zhang, Keqin ^{[8
]}

Cho, Tae-Joon ^{[9
]}

Morhart, Rolf ^{[10
]}

Keen, Richard ^{[11
]}

De Cunto, Carmen L. ^{[12
]}

Friedman, Clive S. ^{[13
]}

Grunwald, Zvi ^{[2
]}

Zasloff, Michael ^{[14
]}

Netelenbos, J. Coen ^{[1
]}

Hsiao, Edward ^{[15
,16
]}

Kaplan, Frederick S. ^{[7
]}

Pignolo, Robert J. ^{[17
]}

Scott, Christiaan ^{[18
,19
]}

Eekhoff, Elisabeth Marelise W. ^{[1
]}

机构：

[1] Univ Amsterdam, locat VU Univ Med Ctr VUMC, Dept Internal Med, Sect Endocrinol Metab,Rare Bone Dis Ctr,Med Ctr,Am, Boelelaan 1117, NL-1081 HV Amsterdam, Netherlands

[2] Thomas Jefferson Univ, Sidney Kimmel Med Coll, Philadelphia, PA USA

[3] Natl Rehabil Ctr Persons Disabil, Tokorozawa, Saitama, Japan

[4] Inst Nacl Rehabil, Dept Genom Med, Mexico City, Mexico

[5] Hosp Israelita Albert Einstein, Res Inst, Sao Paulo, Brazil

[6] Amara Hosp, fDepartment Paediat Orthopaed, Tirupati, India

[7] Univ Penn, Perelman Sch Med, Dept Orthopaed Surg, Penn Med Univ City, Philadelphia, PA USA

[8] Shanghai Tongji Univ, Tongji Hosp, Dept Endocrinol, Shanghai, Peoples R China

[9] Seoul Natl Univ, Div Pediat Orthopaed, Childrens Hosp, Seoul, South Korea

[10] Dept Pediat, Garmisch Partenkirchen, Germany

[11] Royal Natl Orthopaed Hosp NHS Trust, Ctr Metab Bone Dis, London, England

[12] Hosp Italiano Buenos Aires, Potosi, Argentina

[13] Schulich Sch Med & Dent, Pediat Oral Hlth & Dent, London, ON, Canada

[14] Georgetown Univ Hosp, MedStar Georgetown Transplant Inst, Washington, DC USA

[15] Univ Calif San Francisco, Inst Human Genet, Dept Med, Div Endocrinol & Metab,Program Craniofacial Biol, San Francisco, CA USA

[16] Univ Calif San Francisco, Ely & Edythe Broad, Inst Regenerat Med, San Francisco, CA USA

[17] Mayo Clin, Coll Med, Rochester, MN USA

[18] Mayo Clin, Coll Med, Cape Town, South Africa

[19] Univ Ottawa, Div Dermatol & Rheumatol, Ottawa, ON, Canada

来源：

EXPERT OPINION ON PHARMACOTHERAPY | 2025年 / 26卷 / 03期

关键词：

Palovarotene; retinoic acid receptor gamma agonist; fibrodysplasia ossificans progressive; heterotopic ossification; review; outcome treatment trials; RETINOIC ACID SUPPRESSES; HETEROTOPIC OSSIFICATION; GROWTH;

D O I：

10.1080/14656566.2025.2452938

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

IntroductionPalovarotene 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 coveredThe developmental history of palovarotene and an overview of the clinical trials and the regulatory approval journey are discussed by global FOP experts.Expert opinionWhile 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.

引用

页码：291 / 299

页数：9

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