MDA 2026: Duvyzat shows DMD benefits even at lower dose levels
Therapy linked to slower loss of functional muscle tissue in boys
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Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (DMD) regardless of the final dose they received, according to new analyses from a Phase 3 clinical trial.
The treatment also led to a slower loss of functional muscle tissue and a slower replacement of muscle by fat on MRI analyses.
These and other Duvyzat data were presented at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held last week in Orlando, Florida, and online. Duvyzat’s developer, ITF Therapeutics, sponsored the presentations.
“These presentations … reflect our continued commitment to deepening the understanding of long-term treatment with DUVYZAT,” Scott Baver, PhD, vice president, head of global medical affairs, rare diseases for Italfarmaco, the parent company of ITF, said in a company press release. “We are focused on providing people living with DMD, their families, and clinicians with information to support informed treatment decisions regarding DUVYZAT.”
Duvyzat blocks activity of gene-regulating enzymes
Duvyzat is an oral medication that’s approved in the U.S. to treat people with DMD, ages 6 and older. It blocks the activity of gene-regulating enzymes called histone deacetylases, which in turn helps ease inflammation, prevent scarring, and promote muscle regenerationto preserve muscle function for people with DMD.
The therapy’s approval was largely supported by data from the Phase 3 EPIDYS clinical trial (NCT02851797), involving 179 boys with DMD, ages 6 to 17, who were able to walk.
Participants received either twice-daily Duvyzat — at a weight-based dose — or a placebo, along with standard-of-care corticosteroids, for about 1.5 years.
Trial data showed that Duvyzat slowed declines in muscle strength and motor abilities relative to the placebo across multiple functional tests.
Participants on Duvyzat experienced slower functional declines
In a presentation at the MDA meeting, trial investigator Aravindhan Veerapandiyan, MD, of Arkansas Children’s Hospital, investigated whether treatment outcomes differed by the final dose participants received. The talk was titled, “Givinostat Weight-Based Flexible Dosing: Rationale and Efficacy at the Different Doses.”
At the study’s start, participants initiated treatment at a high dose level (dose A). This was reduced, as needed, to two lower dose levels (doses B and C) if side effects occurred. At each dose level, participants were given one of nine specific doses based on their body weight.
After the first 82 participants were enrolled, emerging safety observations led the investigators to eliminate dose level A. As such, subsequent patients began treatment at level B.
Regardless of final dose, people on Duvyzat experienced slower functional declines than those on the placebo, including on the Four-Stair Climb Test, a measure of lower-limb strength, and the North Star Ambulatory Assessment, a more global evaluation of motor abilities.
‘This kind of helps us to … gauge our clinical decision-making when we do decrease this dose in the clinical practice,” Veerapandiyan said.
The scientist, however, pointed out that the nine weight bands used in the EPIDYS trial would be “extremely challenging” to implement in real-world settings.
Despite all of this effort … we have challenges in [clinical] practice in adjusting the doses.
As a result, its developers consolidated these nine weight groups into four broader ones. They also had to make further adjustments to ensure that participants in every weight group would achieve optimal blood levels of the therapy.
This change to the consolidated four-weight-band dosing regimen is now reflected on the therapy’s prescribing label. The physician noted that even with this regimen, patients may still require dose adjustments.
Simulated analyses suggested that in lighter patients, almost 90% would be expected to remain at their original dose, while in the heaviest patients, about 60% might need two dose reductions. That’s similar to what was seen in the EPIDYS trial.
“Despite all of this effort … we have challenges in [clinical] practice in adjusting the doses,” Veerapandiyan said.
A panel of neuromuscular experts has developed guidelines for Duvyzat dose modifications and management of side effects in routine practice. Veerapandiyan presented those recommendations in a poster at the MDA meeting and noted they will soon be published.
Therapy associated with reductions in fat infiltration in leg muscles
In another poster. titled “Givinostat Reduces the Decline of Contractile Cross-Sectional Area and Decreases Fat Infiltration in Patients With Duchenne Muscular Dystrophy,” researchers showed additional muscle-specific data from EPIDYS.
As DMD progresses, muscle tissue wastes away and is replaced by fat. Earlier trial data showed that Duvyzat was associated with significant reductions in fat infiltration in a large thigh muscle compared with the placebo.
In the new analysis, investigators used a specialized MRI technique to assess muscle tissue and fat infiltration across multiple leg muscles in a group of trial participants.
The oral therapy slowed the loss of functional muscle tissue in several leg muscles after about one and 1.5 years. In the same muscles, Duvyzat was associated with reductions in fat infiltration.
“Collectively, these findings … underscore the value of longer-term follow-up and complementary analyses in characterizing the clinical experience of givinostat,” Veerapandiyan said in the press release. “Ongoing evaluation of these data provides important context as clinicians consider appropriate, individualized treatment approaches for patients with DMD,” he added.
Note: The Muscular Dystrophy News Today team is providing live coverage of the 2026 MDA Clinical & Scientific Conference March 8-11 in Orlando, Florida. Go here to see the latest stories from the conference.
