Today, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.

Highlights

• Duvyzat (givinostat) approved by FDA for treating Duchenne Muscular Dystrophy (DMD) in patients aged six and above.
• First nonsteroidal drug approved for all genetic variants of DMD.
• Works as a histone deacetylase (HDAC) inhibitor to reduce inflammation and muscle loss.

Advancements in DMD Treatment

"DMD denies the opportunity for a healthy life to the children it affects. The FDA is committed to advancing the development of new therapies for DMD,” said Emily Freilich, M.D., director of the Division of Neurology 1, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “This approval provides another treatment option to help reduce the burden of this progressive, devastating disease for individuals impacted by DMD regardless of genetic mutation."

Disease Overview

DMD is the most common childhood form of muscular dystrophy, mainly affecting males. It's a rare neurological disorder leading to progressive muscle weakness due to the absence of dystrophin protein. Over time, it causes difficulties in walking, muscle strength, and respiratory problems, often resulting in premature death. However, life expectancy for DMD patients has increased, with some living beyond 30 years.

Efficacy and Study Results

Duvyzat’s efficacy was evaluated in an 18-month phase 3 study. Patients treated with Duvyzat showed significant improvement in muscle function compared to placebo. The mean change in time to climb four stairs was 1.25 seconds for Duvyzat-treated patients versus 3.03 seconds for those receiving placebo.

Additionally, Duvyzat demonstrated less worsening in physical function assessed by the North Star Ambulatory Assessment (NSAA) compared to placebo after 18 months.

Side Effects and Precautions

The most common side effects of Duvyzat include diarrhea, abdominal pain, decreased platelets, nausea/vomiting, increased triglycerides, and fever. Health care providers should monitor platelet counts and triglycerides before and during treatment. Duvyzat should be avoided by patients with certain platelet counts or heart conditions.

Dosage and Administration

The recommended dosage of Duvyzat is determined by the individual’s body weight. It should be administered orally twice daily with food.

Regulatory Status

The FDA granted priority review and fast track designation for Duvyzat. It also received orphan drug and rare pediatric disease designations. The approval was granted to Italfarmaco S.p.A.