Duvyzat conditionally approved by EC to treat Duchenne MD
Recent presentation shows therapy lengthens time to loss of walking ability
The European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating people who are 6 and older with Duchenne muscular dystrophy (DMD).
“This milestone means that a broad range of patients with DMD have access to a new treatment,” Francesco De Santis, president of Italfarmaco Holding and chairman of Italfarmaco Group, said in a company press release. “At Italfarmaco, this achievement reaffirms our focus on advancing therapies that can make a meaningful difference in people’s lives.”
In Europe, “there have been limited approved treatments that address the underlying pathology [disease processes] of DMD across the broad patient population,” said Paola Bettica, MD, PhD, chief medical officer at Italfarmaco Group. “That changes with the approval of Duvyzat, which slows disease progression and preserves muscle function — regardless of the gene mutation — by targeting disease mechanisms.”
Italfarmaco is collaborating with national authorities and distribution partners to facilitate access to the therapy in the 27 member states of the European Union, along with Iceland, Liechtenstein, and Norway. Conditional approval will allow patients to access the medication, while further clinical trials are conducted to confirm Duvyzat’s benefits.
“People living with DMD in Europe have long awaited new therapeutic options that can alter the course of this devastating disease,” Bettica said. “We are committed to working closely with health authorities and the DMD community to ensure timely access to this important new treatment across Europe.”
Duvyzat was approved last year in the U.S., with the U.K. following suit shortly after.
What is Duvyzat?
DMD is a form of muscular dystrophy wherein mutations in the DMD gene make muscles vulnerable to damage. This leads to progressive muscle weakness and wasting, which can result in difficulty walking and breathing, along with other symptoms.
Duvyzat, a small molecule given orally, is intended to slow muscle loss, reduce inflammation, and slow disease progression in people with DMD. It works by blocking enzymes in the histone deacetylase (HDAC) family, which are involved in cellular processes that regulate genetic activity. Specifically, blocking HDAC enzymes increases the production of follistatin, a protein that helps build muscle mass. Reducing the enzymes may increase follistatin’s levels, counteracting the effects of myostatin, a protein that prevents muscle growth and repair.
Data show the therapy lowered inflammation, scarring, and the infiltration of fat into muscles.
Duvyzat’s approvals were based on the Phase 3 EPIDYS trial (NCT02851797), which included 179 boys, ages 6 and older, who were able to walk. The boys received Duzyvat or a placebo twice a day for 18 months, alongside corticosteroids. The primary outcome measure was the time to climb four stairs, a standard assessment where lower scores indicate better function.
After treatment, the Duvyzat group could climb stairs an average of 1.78 seconds faster than the placebo group, a significant difference and clinically meaningful, according to Italfarmaco.
On the North Star Ambulatory Assessment (NSAA), a standard a test of motor function, the Duzyvat participants saw 40% less decline in their overall score over the study period, indicating slower disease progression.
An extension study (NCT03373968) for EPIDYS participants continues to monitor safety and efficacy. Results presented in March showed that boys on Duvyzat in the extension study lost the ability to walk at a median age of 18.1, compared to 15.2 in a control natural history population. Side effects of Duvyzat have included diarrhea, abdominal pain, increased blood fat levels, and low platelet counts.
The company is running other clinical trials in a broader group of participants. A Phase 3 study (NCT05933057), recruiting at sites in Europe and Canada, is testing the medication against a placebo in boys with DMD, ages 9-17, who can’t walk. A Phase 2 study (NCT06769633), also in Europe, is testing the medication in boys, ages 2-5.
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