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givinostat (Duvyzat)
Indications:
- treatment of Duchenne Muscular Dystrophy (DMD) in boys >= 6 years of age
Dosage:
- 10 mg/mL PO BID (dose flexible, based on weight)
- reduce dose if not tolerated
Mechanism of action:
- histone deacetylase (HDAC) inhibitor
- prevents gene translation through changing the 3D folding of cell DNA
- inhibiting HDACs promotes muscle repair, reduces inflammation, & decreases both muscle fibrosis & fat accumulation in patients
General
metabolic agent (metabolic modifier)
enzyme inhibitor
References
- FDA NEWS RELEASE. March 21, 2024
FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy
https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
- Shaw ML
Givinostat Receives Full FDA Approval for Duchenne Muscular Dystrophy
American Journal of Managed Care (AJMC). 2024. March 21.
https://www.ajmc.com/view/givinostat-receives-full-fda-approval-for-duchenne-muscular-dystrophy
- Mercuri E, Vilchez JJ, Boespflug-Tanguy O et al
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS):
a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Lancet Neurol. 2024 Apr;23(4):393-403.
PMID: 38508835 Clinical Trial.
https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(24)00036-X/fulltext