Duvyzat Approved for Duchenne Muscular Dystrophy

On March 21, 2024, the U.S. Food and Drug Administration (FDA) approved Duvyzat^™ (givinostat – Italfarmaco). Duvyzat, a histone deacetylase (HDAC) inhibitor, is indicated for treating Duchenne Muscular Dystrophy (DMD) in patients aged six years and older. Before starting therapy, the patient’s platelet count and triglyceride levels should be evaluated. Also, platelet counts should be monitored while therapy continues. The recommended dosage of Duvyzat, which depends on body weight, should be administered orally twice daily with food. Italfarmaco’s subsidiary, ITF Therapeutics, will make Duvyzat available in the U.S. Launch and pricing information is not yet available. You can find the complete prescribing information here.

At a Glance

• Brand Drug: Duvyzat (givinostat)
• Manufacturer: Italfarmaco S.p.A./ITF Therapeutics
• Date Approved: March 21, 2024
• Indication:  treatment of DMD in patients six years of age and older
• Dosage Forms Available: bottles containing 140mL (8.86mg/mL) of oral suspension
• Launch Date: Not yet available
• Estimated Annual Cost: Not yet available
• DMD is a rare genetic condition that affects around 10,000 patients, almost exclusively boys and young men, in the United States.
• In DMD, a mutation in the gene for dystrophin, a muscle protein, causes progressive muscle wasting — typically beginning with muscles in the shoulders and thighs, then spreading gradually to other muscles, including the heart. Eventually, DMD patients need to use wheelchairs and ventilators. Currently, few patients live beyond 30 years of age.
• The precise mechanism of Duvyzat’s activity is not fully understood. In patients who have DMD, dystrophin deficiency is believed to increase histone deacetylase (HDAC) enzyme activity, which can interfere with muscle regeneration and repair. Duvyzat, as an HDAC inhibitor, blocks the effects of these enzymes.
• In its clinical trial, all participants received a standard-of-care steroid regimen throughout the study. After 18 months, patients who also received Duvyzat showed statistically less functional decline compared to placebo. Patients who received the drug on average took 1.23 seconds longer to climb four stairs, versus about 3 seconds longer for those receiving placebo.
• The most common adverse events (AEs) reported in the trial were diarrhea and vomiting.
• Sarepta’s gene therapy, Elevidys (delandistrogene moxeparvovec-rokl), received conditional (accelerated) approval in June 2023 for treating certain boys, aged four to five years old, who have DMD. The company is seeking to gain full approval for treating all patients for DMD. The FDA is expected to rule on this application by June 21, 2024.
• Sarepta also manufactures three other drugs that treat symptoms of different DMD variations. Exondys 51 (eteplirsen) was launched in the U.S. in September 2016 for patients who have DMD mutations amenable to exon 51 skipping – about 13% of patients. Vyondys 53 (golodirsen) was released in December 2019 for the estimated 8% of patients who have a confirmed mutation amenable to exon 53 skipping. Amondys 45 (casimersen), FDA approved in February 2021, treats patients who have DMD that has tested positive for a mutation that is amenable to exon 45 skipping. All three received Accelerated Approval, so clinical testing is continuing for them, as well as for Elevidys.
• In August 2020, Viltepso^® (viltolarsen – NS Pharma) was FDA approved – also under the FDA’s Accelerated Approval program – for treating DMD amenable to exon 53 skipping.
• Emflaza^™ (deflazacort), which is made by Marathon Pharmaceuticals, is indicated to treat DMD symptoms for patients who are five years old and older. An oral corticosteroid decreases both immune and inflammatory activity to relieve symptoms of DMD and also to delay the progression of the disease. The first generic Emflaza tablets were launched in February 2024.
• Approved under the FDA’s Priority Review and Fast Track processes, Duvyzat also has Orphan Drug and Rare Pediatric Disease designations.