Duvyzat: A New Treatment for Duchenne Muscular Dystrophy Approved on March 21, 2024
What is it prescribed for?
DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.
What is the name of the drug and what does it do?
Duvyzat (pronounced doo-VY-zat), (generic name: givinostat), is a medication indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. It helps improve muscle function and slow the progression of the disease.
How does it work?
Duvyzat is a histone deacetylase (HDAC) inhibitor. HDACs are enzymes that modify proteins called histones, which help regulate the expression of genes. By inhibiting HDACs, Duvyzat can alter gene expression in muscle cells, leading to increased muscle strength and decreased muscle degeneration, which are characteristic of DMD.
What did the research discover?
Clinical trials have shown that Duvyzat is effective in improving muscle function in patients with DMD. In a randomized, double-blind, placebo-controlled 18-month study, patients treated with Duvyzat showed statistically significant less decline in the 4-stair climb time compared to placebo.
Additionally, patients treated with Duvyzat experienced less worsening on the North Star Ambulatory Assessment (NSAA) compared to placebo.
What are some of the side effects?
- Diarrhea
- Abdominal pain
- Thrombocytopenia
- Nausea/vomiting
- Hypertriglyceridemia
- Pyrexia (fever)
- Myalgia (muscle pain)
- Rash
- Arthralgia (joint pain)
- Fatigue
- Constipation
- Decreased appetite
What are the dosage recommendations and how is it prescribed?
The recommended dosage of Duvyzat is based on body weight and is administered orally twice daily with food. The specific dosage for different weight categories is as follows:
- 10 kg to less than 20 kg: 22.2 mg twice daily (2.5 mL twice daily)
- 20 kg to less than 40 kg: 31 mg twice daily (3.5 mL twice daily)
- 40 kg to less than 60 kg: 44.3 mg twice daily (5 mL twice daily)
- 60 kg or more: 53.2 mg twice daily (6 mL twice daily)
Dosage modifications may be needed for adverse reactions such as decreased platelet counts, diarrhea, increased triglycerides, or QTc prolongation. It is essential to shake the oral suspension well before each dose and to use the provided oral syringe for accurate measurement.
Source:
Italfarmaco S.A. Duvyzat (givinostat) oral suspension, for oral use. Prescribing Information. 2024. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/217900s000lbl.pdf
