Rollout expected by fall in US for new DMD therapy Duvyzat

Treatment is approved for patients regardless of disease-causing mutation

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

Speech bubbles show the main points of conversation between two people talking while seated side by side at a conference table.

ITF Therapeutics — which will be responsible for marketing Duvyzat (givinostat), Italfarmaco’s newly approved Duchenne muscular dystrophy (DMD) therapy, in the U.S — expects the oral medication to be available to eligible adults and children by this fall.

For ITF, the U.S.-based rare disease division of Italfarmaco, last month’s approval of Duvyzat by the U.S. Food and Drug Administration (FDA) offers new choices for DMD patients.

“We are humbled to serve the DMD community and thrilled for the opportunity to bring a new treatment option forward for this challenging disease,” Matt Trudeau, general manager of ITF Therapeutics, said in a written Q&A with Muscular Dystrophy News Today.

“Following the FDA approval, our team at ITF Therapeutics is working to complete all the steps necessary to make Duvyzat available to appropriate patients in the U.S. as quickly as possible,” Trudeau said, anticipating a launch “in the third quarter of 2024.”

“In the meantime,” Trudeau added, “we will be diligently working to ensure the support required to make Duvyzat available to the DMD community will be in place and optimized to serve patients, their families, and healthcare providers.”

Unlike other treatments for Duchenne, Duvyzat is indicated for all patients ages 6 and older, regardless of the disease-causing mutation they carry.

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Oral therapy approved last month for all patients, ages 6 and older

Until now, treatments for Duchenne have been designed to target specific DMD-causing mutations, making their utility limited to patients with specific gene alterations. Duvyzat was created to treat DMD patients with any disease-causing mutation.

The therapy is designed to block enzymes of the histone deacetylase (HDAC) family. This switches on the gene coding for follistatin, which helps to build muscle mass by acting against myostatin, a protein that limits muscle growth and promotes protein breakdown.

In DMD, “the impairment of muscle repair causes chronic inflammation, muscle degeneration and eventual replacement of muscle tissue with scar and fatty tissues. All of this damage eventually translates to loss of function and physical ability,” Trudeau said.

The FDA’s decision to approve Duvyzat, which comes as an oral suspension to be taken by mouth at a dose that depends on body weight, was based on data from the Phase 3 EPIDYS study (NCT02851797). That clinical trial involved 179 boys with DMD, ages 6 to 17, who were able to walk.

All boys continued to receive standard of care with a stable dose of corticosteroids. In treatment spanning 1.5 years, Duvyzat slowed muscle function decline, with boys on Duvyzat taking an average of 1.78 fewer seconds to climb four stairs than those on a placebo.

Moreover, boys on Duvyzat built up about 30% less fatty tissue in muscle compared with those on the placebo.

“By slowing muscle function decline and fat buildup, our goal is to help preserve the ability to perform certain daily life activities,” Trudeau said.

While pivotal data came from boys who were able to walk, Duvyzat also may be used by patients who cannot walk, Trudeau noted. He disclosed plans to begin testing Duvyzat in younger patients soon.

“We are excited to share that an open-label study in DMD patients [younger than] 6 years of age is planned in 2024,” Trudeau said.

“We will keep the community informed when we start recruiting for this clinical trial,” he added.

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An ongoing open-label study (NCT03373968), sponsored by Italfarmaco, is looking into the long-term safety of Duvyzat.

In addition, the company is sponsoring a global Phase 3 clinical trial called ULYSSES (NCT05933057) that will run through 2028. It expects to recruit as many as 138 boys with DMD, ages 9 to 17, who have lost the ability to walk.

“We will continue to research and further quantify the potential benefits of Duvyzat. We hope that both of these studies provide more insights regarding how Duvyzat can help slow disease progression in patients with DMD,” Trudeau said.

We will offer a range of services to support access to Duvyzat. … We are in the process of gathering feedback from the DMD community to ensure the programs and services we develop meet the needs of patients and their families.

The most common side effects reported with Duvyzat include diarrhea, abdominal pain, low numbers of platelets in the blood, nausea, and vomiting. High levels of triglycerides, a type of fat, in the blood and fever also have been reported.

“We will offer a range of services to support access to Duvyzat,” Trudeau said. “We are in the process of gathering feedback from the DMD community to ensure the programs and services we develop meet the needs of patients and their families.”

Duvyzat is under regulatory review in Europe, where it holds orphan drug designation as a treatment for DMD. The medication also is being tested for Becker muscular dystrophy, but clinical development for this type of muscular dystrophy is still “in the early stages,” Trudeau said.