Duvyzat, a Nonsteroidal Treatment for Duchenne Muscular Dystrophy, Gets FDA Approval

The Food and Drug Administration (FDA) has approved Duvyzat^™ (givinostat) for the treatment of patients 6 years of age and older with Duchenne muscular dystrophy (DMD).

The approval was based on data from the randomized, double-blind, placebo-controlled phase 3 EPIDYS study (ClinicalTrials.gov Identifier: NCT02851797). The study assessed the efficacy and safety of givinostat, a histone deacetylase inhibitor, in male patients 6 years of age and older with DMD who were ambulatory and on a stable dosage of corticosteroids. Study participants were randomly assigned to receive either givinostat (n=118) or placebo (n=61).

The primary endpoint was the change from baseline to month 18 in 4-stair climb time, a measure of muscle function that tests the time it takes to climb 4 stairs. The primary analysis population included 81 patients in the Duvyzat arm and 39 patients in the placebo arm.

Results showed treatment with givinostat was associated with statistically significant less decline in the 4-stair climb time compared with placebo (treatment difference from placebo, -1.78 seconds [95% CI, -3.46, -0.11]; P =.037). 

While not statistically significant, patients treated with givinostat experienced less worsening than those in the placebo group on the North Star Ambulatory Assessment, a 17-item rating scale used to measure functional motor abilities in ambulant children with DMD.

“There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients,” said Craig M. McDonald, MD, Professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health and investigator for the EPIDYS trial. “Duvyzat’s unique mechanism of action has shown a positive risk/benefit profile and the ability to delay disease progression, supporting its potential to become a key component of the standard of care for people living with DMD.”

The most common adverse reactions reported with Duvyzat were diarrhea, abdominal pain, thrombocytopenia, nausea/vomiting, hypertriglyceridemia, and pyrexia. 

Prior to initiating treatment, baseline platelet counts and triglycerides should be obtained and monitoring during treatment is recommended. Additionally, for patients with underlying cardiac disease or those on concomitant medications that prolong the QTc interval, an ECG should be performed and repeated as clinically indicated.

The recommended dosage of Duvyzat is based on body weight and administered orally twice daily with food. Dosage modification may be required for decreased platelet counts, diarrhea, increased triglycerides, or QTc prolongation.

Duvyzat is supplied as an oral suspension containing 8.86mg of givinostat per mL in a 140mL bottle. The product is expected to be available in the third quarter of 2024.