FDA’s Approval of Duvyzat (givinostat) Offers New Hope to People Living with Duchenne Muscular Dystrophy

On March 21, the  U.S. Food and Drug Administration gave final approval to Duvyzat (givinostat), a new treatment for children and adolescents living with Duchenne muscular dystrophy (DMD).

In clinical trials, the treatment, a histone deacetylase (HDAC) inhibitor, was shown to slow the progression of this disease. The Muscular Dystrophy Association, who funded the foundational research which played a significant role in the development of Duvyzat (givinostat), announced the news in a press release.

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.

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 HDAC inhibitors (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. Pier Lorenzo Puri group has performed the studies that led to the discovery of HDAC inhibitors for treatment of DMD.

Duchenne muscular dystrophy (DMD) is a progressive genetic muscular disease that is caused by the lack of an important membrane-stabilizing protein called “dystrophin.” People with this condition have progressive muscle degeneration and weakness, with symptoms usually appearing between the ages of two and three.

How Duvyzat (givinostat) Works

Like other HDAC inhibitors, Givinostat changes the gene expression in cells by altering the three-dimensional folding of DNA. Studies have shown that this process can slow the progression of DMD. Givinostat will be made available in the United States by ITF Therapeutics.

“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.”

MDA’s Legacy in Duchenne Muscular Dystrophy Research

Since our inception, MDA has invested over $229 million in research for Duchenne and Becker muscular dystrophy.

The discovery of the dystrophin gene in the 1980s by MDA-funded research marked the beginning of our understanding of Duchenne and Becker muscular dystrophy. This discovery has been foundational in the development of treatments like Duvyzat (givinostat).

Today’s celebration of this groundbreaking treatment is only possible because of the dedication and generosity of our community of supporters. Together, we are turning research into reality for people living with neuromuscular diseases.

At its founding the MDA was the one researcher in the U.S. focused on muscular dystrophy. Today, there are multiple therapies available to treat the disease, and a tremendous pipeline of more treatment options coming.

MDA family members, Jessica and Christopher Curran with their children Kyle, William, Conner.

“Being part of the Muscular Dystrophy Association is everything. From the MDA Care Center Network and MDA 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,” said Jessica Curran, MDA family member whose son, Conner, who lives with DMD.

“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.

“When my son Conner was diagnosed nine years ago, there were ten 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,” Curran said.