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Muscular Dystrophy Association’s Funding of Foundational Research Leads to New FDA Approved Treatment Duvyzat (givinostat) for Duchenne Muscular Dystrophy

NEW YORK, March 21, 2024 – The Muscular Dystrophy Association’s funding of foundational research leads to new drug approval by the US Food and Drug Administration (FDA) of Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor to treat children and adolescents living with Duchenne muscular dystrophy (DMD). This drug changes gene expression in cells by altering the three-dimensional folding of DNA and has shown that it can slow DMD progression. Duvyzat (givinostat) will be made available in the United States by ITF Therapeutics.

Results from a phase 3 clinical trial of Duvyzat (givinostat) recently published in The Lancet demonstrated a significantly smaller decline in stair climbing ability in treated patients compared to a placebo control group.

"The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA. “As more drugs are approved for DMD we look forward to understanding if and how they might be used in combination.”

Pier Lorenzo Puri’s group based at Sanford Burnham Prebys Medical Discovery Institute in San Diego performed the studies that led to the discovery of HDAC inhibitors (HDACi) for treatment of DMD since 2004. These studies were performed in close collaboration with the lab Puri led in Rome in Scientific Park of Castel Romano (2002-2007) and at Fondazione Santa Lucia (2008-2018).

“Muscular Dystrophy Association’s funding was fundamental for the discovery of HDACi in the treatment of Duchenne muscular dystrophy. We are grateful to MDA to have enabled studies that support the important pre-clinical evidence that HDACi exerted beneficial effects in the mouse model of DMD. These studies provided the initial experimental evidence that led me to contact Italfarmaco to test givinostat, as a HDACi that was already in clinical trial for other diseases and could have been easily repurposed for DMD,” said Pier Lorenzo Puri, M.D., Sanford Burnham Prebys Medical Discovery Institute.

For decades, MDA has awarded many research grants to fund investigators worldwide and their promising early-stage science. These grants support the foundational discoveries that enable future clinical translation, such as with Duvyzat (givinostat).

“Since the way Duvyzat (givinostat) works is different from other treatments for Duchenne muscular dystrophy, the combination of this new drug with other therapies may further improve the lives of our patients living with Duchenne muscular dystrophy,” said Barry Byrne, M.D., Ph.D., Chief Medical Advisor, MDA and the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida.

“We’re excited to celebrate this additional treatment option for people living with Duchenne muscular dystrophy. This approval demonstrates the FDA’s understanding of the importance of slowing disease progression in diseases like Duchenne, and how meaningful that is to the community. We are proud to partner with the FDA to help speed the approval of new therapies that slow disease progression and ensure the voice of patients and their families is heard,” said Paul Melmeyer, Vice President, Public Policy and Advocacy, MDA.

“Being part of the Muscular Dystrophy Association is everything. From the MDA Care Center Network and Resource Center to the industry development, educational resources, and advocacy and so much more, because of MDA when people are diagnosed, they have such a wide range of resources. When my son Conner was diagnosed nine years ago, there were 10 drugs in the pipeline for DMD and steroids possibly if you chose to do that -- and really not much else. With this new FDA approval, it's amazing because of the MDA, they've funded the seed research, for a lot of the drugs that are now in the pipeline or already approved. And parents have choices. It is more than fair to say that the Muscular Dystrophy Association has improved lives. My son is an example,” said Jessica Curran, MDA family member and mother of Conner who lives with Duchenne muscular dystrophy.

About Duchenne muscular dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is a rare and progressive genetic muscle disease caused by the lack of a critical membrane-stabilizing protein called dystrophin, which results in muscle degeneration and weakness. DMD symptom onset is in early childhood, usually between ages 2 and 3. The disease primarily affects boys, but in rare cases it can affect girls. In Europe and North America, the prevalence of DMD is approximately 6 per 100,000 individuals. Doctors often diagnose the disease between ages 3 and 6 when children show early signs of significant muscle weakness, such as delayed ability to sit, stand, or walk and difficulties learning to speak. Over time, DMD becomes fatal as muscle degeneration disrupts lung and heart function.

About Muscular Dystrophy Association

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.