Duvyzat now available to DMD patients 6 and older in US
Global Phase 3 trial recruiting boys, ages 9 to 17, with DMD
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Duvyzat (givinostat), a newly approved treatment for Duchenne muscular dystrophy (DMD), is now available in the U.S., according to an announcement from ITF Therapeutics, which will be selling the therapy.
“Following the FDA [Food and Drug Administration] approval of Duvyzat, our team has been focused on making this new treatment option available to the DMD community as rapidly as possible,” Matt Trudeau, president of ITF, said in a company press release.
Patients and caregivers interested in starting on the therapy are advised to contact their clinicians to get the process going.
“We have also deployed field teams and easy-to-navigate communication platforms and services to provide patients, caregivers, and clinicians with the information and resources they need to make informed decisions about treatment with Duvyzat for DMD,” Trudeau said.
Patient support program available
Among the resources available to patients, ITF is offering a support program called ITF ARC, which “offers helpful information in areas including navigating insurance coverage,” Trudeau said. Additional resources can be found on the therapy’s website or by phone at 1-855-448-3272.
“We are very grateful for the guidance that we received from DMD advocacy leaders throughout the development of ITF ARC,” Trudeau said. “Their input has been fundamental in helping us design the suite of services we are offering to the community.”
News of Duvyzat’s approval was met with joy from the DMD community. Parent Project Muscular Dystrophy said in a statement it “is thrilled to see another approved therapeutic option for Duchenne become available to patients in the U.S.”
Duvyzat is designed to block the activity of histone deacetylases, which are a group of enzymes that help to control genetic activity in cells. By modulating these enzymes, the therapy switches on the gene coding for follistatin to help build muscle mass, thus acting to slow the progressive muscle weakness and wasting that characterizes DMD.
Duvyzat approved by FDA earlier this year for DMD patients 6 and older
Duvyzat was approved by the U.S. FDAÂ earlier this year for DMD patients 6 and older. It is notably the first non-steroid treatment for DMD to be authorized for patients with any type of disease-causing mutation.
FDA approval of Duvyzat was based on data from a Phase 3 clinical trial called EPIDYS (NCT02851797), which tested the therapy against a placebo in more than 170 boys with DMD. Results showed that, after 1.5 years of treatment, patients given Duvyzat had significantly slowed loss of motor function.
Another Phase 3 trial, ULYSSES (NCT05933057), is testing the therapy in boys, ages 9 to 17, with DMD who have lost the ability to walk. That study is still recruiting patients at sites in Canada, Belgium, France, Italy, the Netherlands, and the U.K. Patients who finish trials of Duvyzat can opt to enroll in an open-label study (NCT03373968) tracking long-term outcomes with the therapy.
Duvyzat is being considered for approval in the European Union.
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