Nonsteroidal Drug for Duchenne Launches with a $700,000 Price Tag

ITF Therapeutics has launched Duvyzat (givinostat) to treat patients with Duchenne muscular dystrophy (DMD) who are six years of age and older.

Duchenne is a rare genetic disease that is characterized by a mutation in the dystrophin gene. It causes the muscles in the body to become weak and damaged over time and is eventually fatal. Duchenne affects about 1 in 3,500 to 5,000 boys born worldwide

Duvyzat is the first nonsteroidal oral drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and loss of muscle. Duvyzat was developed by Milan-based Italfarmaco in partnership with Telethon and Duchenne Parent Project (Italy). It is being marketed in the United States by ITF Therapeutics, a new subsidiary of Italfarmaco.

Dosing of Duvyzat is based on weight, but the average annual cost of Duvyzat is $700,000 based on the typical patient studied in the clinical trial, according to an ITF company spokesperson.

The company is offering ITF ARC (Access, Resources and Care), which includes services to help patients and families navigate through insurance challenges, personalized pharmacist support, financial and access assistance for eligible patients, educational materials, and other resources.

Commercially insured patients may be eligible for a $0 copay. The terms indicate there is maximum benefit but that is not shown. The terms indicate a limit may be limit may if “ITF Therapeutics determines that a third party has implemented a program that adjusts patient cost-sharing obligations based on the availability of support under the program and/or excludes the assistance provided under the program from counting towards the patient’s deductible or out-of-pocket cost limitations.”

The approval of Duvyzat in March 2024 is based on the results of the pivotal phase 3 EPIDYS trial, which enrolled 179 boys six years of age or older who received either Duvyzat twice daily or placebo, in addition to glucocorticosteroid treatment. The study met its primary endpoint demonstrating that patients on Duvyzat showed a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment.

The most common side effects of Duvyzat are diarrhea, abdominal pain, a decrease in platelets — which can lead to increased bleeding — nausea/vomiting, an increase in triglycerides (a type of fat in the body) and fever. The majority of adverse effects were mild to moderate in severity. Results from this study were published in The Lancet Neurology in March 2024.

The prescribing information includes warnings about evaluating patients’ platelet counts and triglycerides before prescribing Duvyzat. Additionally, Duvyzat may also cause QTc prolongation, which can increase the risk for irregular heartbeats.