Duvyzat recommended for EU approval as DMD treatment
Decision on Italfarmaco's disease-modifying therapy expected in July
The Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency known as CHMP for short, has recommended the approval of Duvyzat (givinostat) as a treatment for people with Duchenne muscular dystrophy (DMD), ages 6 and older, who are able to walk.
The European Commission will review this recommendation, with a decision expected in July. CHMP’s recommendations are not binding, but are usually followed.
If the DMD treatment is approved, developer Italfarmaco said it will “work closely with local authorities to facilitate rapid access” to Duvyzat across the European Union, per a company press release.
According to Eugenio Mercuri, MD, a professor of pediatric neurology at the Catholic University in Rome, “the urgent need for disease-modifying therapies in Duchenne cannot be overstated, and this CHMP recommendation marks a critical step forward.”
Duvyzat was approved in the U.S. last year, with a similar decision in the U.K. following soon after.
DMD treatment backed for patients 6 and older who can walk
In muscular dystrophy (MD), genetic mutations cause progressive weakening of the body’s muscles, which leads to symptoms such as difficulty walking and breathing. DMD, the most common form of muscular dystrophy, is caused by mutations in the DMD gene that make muscles more susceptible to damage. More boys than girls are affected by DMD.
Duvyzat, an oral therapy, blocks enzymes in the histone deacetylase family, which help regulate the genetic activity of cells. Data have shown that Duvyzat can help lower inflammation, and ease scarring and fat infiltration into muscles.
“Its unique mechanism of action represents an important addition to the treatment landscape, offering new hope that Duvyzat could become a foundational therapy for those living with Duchenne,” Mercuri said.
CHMP’s decision to recommend the therapy’s approval is based on data from the Phase 3 EPIDYS clinical trial (NCT02851797), which tested the effects of Duvyzat in boys ages 6 to 17 who were able to walk and taking corticosteroids for DMD. Over nearly 1.5 years of treatment, Duvyzat slowed motor function decline relative to a placebo.
At the end of the study period, boys who received the therapy were able to climb four stairs more quickly than patients taking the placebo (1.78 seconds difference). Scores of the treated boys on the North Star Ambulatory Assessment, a test of motor function, also declined 40% less in the Duvyzat group.
Together with the DMD community, we welcome the CHMP’s opinion, which moves us closer to making Duvyzat available to eligible patients living with Duchenne in the EU.
As for safety, 95% of side effects were mild to moderate in severity. The most common side effects were decreased platelet counts, increased blood fat levels, diarrhea, and abdominal pain.
An extension study (NCT03373968) that has been monitoring participants to assess long-term outcomes has shown that use of Duvyzat helped preserve the ability to walk and climb stairs. Treatment also slowed declines in lung function after boys lost the ability to walk.
“Together with the DMD community, we welcome the CHMP’s opinion, which moves us closer to making Duvyzat available to eligible patients living with Duchenne in the EU,” said Francesco De Santis, Italfarmaco’s chairman. “The positive decision supports bringing this novel treatment to patients while we continue to generate additional clinical evidence.”
Additional studies testing Duvyzat in broader patient populations
To further assess the therapy and potentially broaden the patient population eligible for Duvyzat, Italfarmaco is also running a Phase 3 study (NCT05933057) to test the medication in boys ages 9 to 17 who cannot walk. That placebo-controlled study, expected to run through 2028, is still recruiting at multiple sites in Europe and Canada.
An open-label Phase 2 study (NCT06769633) — meaning both participants and researchers know the medication patients are receiving — that’s testing the DMD treatment candidate in younger boys, also is recruiting at sites in Europe. It will involve participants ages 2 to 5.
Paolo Bettica, MD, PhD, chief medical officer at Italfarmaco, called the CHMP recommendation “a validation of Duvyzat’s therapeutic potential.”
“We are profoundly grateful to the families, caregivers, and patient communities whose engagement and advocacy have been instrumental in reaching this significant achievement,” Bettica added.
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