The Duchenne muscular dystrophy (DMD) community has a reason to celebrate. According to a recent news release, the FDA recently approved Duvyzat (givinostat) for people ages six and older who are living with Duchenne muscular dystrophy.

Duvyzat is a histone deacetylase (HDAC) inhibitor administered via oral suspension. Developed by global biopharmaceutical company Italfarmaco S.p.A., Duvyzat is an investigational therapy that works to module dysregulated HDAC activity. Dysregulated HDAC activity can be linked to a lack of dystrophin, which is characteristic of Duchenne muscular dystrophy. Therefore, Duvyzat works to reduce this overactivity and thus, reduce muscle damage. Debra Miller, CureDuchenne Founder and CEO, shares:

“We are pleased that the FDA approved Duvyzat (givinostat) for individuals with Duchenne age six and older. This adds to the list of approved treatments for families facing this devastating disease and is an important step forward in accelerating transformative treatments for everyone independent of their genetic mutation.”

Duvyzat’s approval is based on data from the Phase 3 EPIDYS study. 179 ambulant (able to walk) boys with DMD enrolled. During the study, participants were given either a placebo or Duvyzat twice each day, as well as a glucocorticosteroid. The research team found that:

• Children treated with Duvyzat showed improvements in ambulation, mobility, and climbing stairs.
• Duvyzat was safe and well-tolerated. While some children did experience side effects, there were mild or moderate. Potential side effects include nausea and vomiting, thrombocytopenia (low platelet counts), abdominal pain, diarrhea, fever, and increased fat in the blood.

Learn more about Duvyzat here.

What to Know: Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle weakness and degeneration. Inherited in an X-linked recessive pattern, DMD gene mutations cause low or missing dystrophin. Dystrophin is a protein that maintains the structure and strength of muscle fibers. Without dystrophin, the muscle become damaged over time. DMD primarily affects males, though females can be affected.

Symptoms of Duchenne muscular dystrophy often appear in early childhood between ages three and five. These typically manifest as:

• Muscle weakness beginning in the legs, pelvis, and thighs that progresses to the rest of the body
• Frequent tripping and falling
• Difficulty standing, moving positions, climbing stairs, or walking
• Learning disabilities
• Scoliosis (abnormal spinal curvature)
• Fatigue
• Calf hypertrophy (enlarged calves)
• Respiratory difficulties due to chest muscle weakness