Givinostat: First Approval

被引：26

作者：

Lamb, Yvette N. ^{[1
]}

机构：

[1] Springer Nat, Private Bag 65901, Auckland 0754, New Zealand

来源：

DRUGS | 2024年 / 84卷 / 07期

关键词：

DUCHENNE MUSCULAR-DYSTROPHY; BOYS; SAFETY;

D O I：

10.1007/s40265-024-02052-1

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Givinostat (DUVYZAT (TM)), an orally available histone deacetylase inhibitor, is being developed by Italfarmaco for the treatment of muscular dystrophy and polycythemia vera. Givinostat received its first approval on 21 March 2024, in the USA, for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. Approval was based on the results of the multinational phase III EPIDYS trial, in which givinostat recipients showed less decline than placebo recipients in the time taken to perform a functional task. Givinostat represents the first nonsteroidal treatment for DMD to be approved for use in patients irrespective of the specific genetic variant underlying their disease. Givinostat is available as an oral suspension to be administered twice daily with food. The recommended dosage is based on the body weight of the patient. In the EU, regulatory review of givinostat in DMD is currently underway. This article summarizes the milestones in the development of givinostat leading to this first approval for DMD.

引用

页码：849 / 856

页数：8

共 28 条

[1]

[Anonymous], ClinicalTrials.gov

[2] Histological effects of givinostat in boys with Duchenne muscular dystrophy [J].

Bettica, Paolo ;

Petrini, Stefania ;

D'Oria, Valentina ;

D'Amico, Adele ;

Catteruccia, Michela ;

Pane, Marika ;

Sivo, Serena ;

Magri, Francesca ;

Brajkovic, Simona ;

Messina, Sonia ;

Vita, Gian Luca ;

Gatti, Barbara ;

Moggio, Maurizio ;

Puri, Pier Lorenzo ;

Rocchetti, Maurizio ;

De Nicolao, Giuseppe ;

Vita, Giuseppe ;

Comi, Giacomo P. ;

Bertini, Enrico ;

Mercuri, Eugenio .

NEUROMUSCULAR DISORDERS, 2016, 26 (10) :643-649

[3] Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study [J].

Comi, Giacomo P. ;

Niks, Erik H. ;

Vandenborne, Krista ;

Cinnante, Claudia M. ;

Kan, Hermien E. ;

Willcocks, Rebecca J. ;

Velardo, Daniele ;

Magri, Francesca ;

Ripolone, Michela ;

van Benthem, Jules J. ;

van de Velde, Nienke M. ;

Nava, Simone ;

Ambrosoli, Laura ;

Cazzaniga, Sara ;

Bettica, Paolo U. .

FRONTIERS IN NEUROLOGY, 2023, 14

[4] Preclinical Studies in the mdx Mouse Model of Duchenne Muscular Dystrophy with the Histone Deacetylase Inhibitor Givinostat [J].

Consalvi, Silvia ;

Mozzetta, Chiara ;

Bettica, Paolo ;

Germani, Massimiliano ;

Fiorentini, Francesco ;

Del Bene, Francesca ;

Rocchetti, Maurizio ;

Leoni, Flavio ;

Monzani, Valmen ;

Mascagni, Paolo ;

Puri, Pier Lorenzo ;

Saccone, Valentina .

MOLECULAR MEDICINE, 2013, 19 :79-87

[5] The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD [J].

Duong, Tina ;

Canbek, Jennifer ;

Birkmeier, Marisa ;

Nelson, Leslie ;

Siener, Catherine ;

Fernandez-Fernandez, Alicia ;

Henricson, Erik ;

McDonald, Craig M. ;

Gordish-Dressman, Heather .

JOURNAL OF NEUROMUSCULAR DISEASES, 2021, 8 (06) :939-948

[6] A phase II study of Givinostat in combination with hydroxycarbamide in patients with polycythaemia vera unresponsive to hydroxycarbamide monotherapy [J].

Finazzi, Guido ;

Vannucchi, Alessandro M. ;

Martinelli, Vincenzo ;

Ruggeri, Marco ;

Nobile, Francesco ;

Specchia, Giorgina ;

Pogliani, Enrico Maria ;

Olimpieri, Odoardo Maria ;

Fioritoni, Giuseppe ;

Musolino, Caterina ;

Cilloni, Daniela ;

Sivera, Piera ;

Barosi, Giovanni ;

Finazzi, Maria Chiara ;

Di Tollo, Silvia ;

Demuth, Tim ;

Barbui, Tiziano ;

Rambaldi, Alessandro .

BRITISH JOURNAL OF HAEMATOLOGY, 2013, 161 (05) :688-694

[7] Population pharmacokinetic-pharmacodynamic analysis of givinostat [J].

Fiorentini, Francesco ;

Germani, Massimiliano ;

Del Bene, Francesca ;

Pellizzoni, Cinzia ;

Cazzaniga, Sara ;

Rocchetti, Maurizio ;

Bettica, Paolo .

EXPERT OPINION ON DRUG METABOLISM & TOXICOLOGY, 2023, 19 (04) :229-238

[8] Pharmacokinetics, Safety and Inducible Cytokine Responses during a Phase 1 Trial of the Oral Histone Deacetylase Inhibitor ITF2357 (Givinostat) [J].

Furlan, Antonio ;

Monzani, Valmen ;

Reznikov, Leonid L. ;

Leoni, Flavio ;

Fossati, Gianluca ;

Modena, Daniela ;

Mascagni, Paolo ;

Dinarello, Charles A. .

MOLECULAR MEDICINE, 2011, 17 (5-6) :353-362

[9] The histone deacetylase inhibitor ITF2357 selectively targets cells bearing mutated JAK2V617F [J].

Guerini, V. ;

Barbui, V. ;

Spinelli, O. ;

Salvi, A. ;

Dellacasa, C. ;

Carobbio, A. ;

Introna, M. ;

Barbui, T. ;

Golay, J. ;

Rambaldi, A. .

LEUKEMIA, 2008, 22 (04) :740-747

[10]

Hoffman EP, 2020, HANDB EXP PHARMACOL, V261, P25, DOI 10.1007/164_2019_256

← 1 2 3 →