ITF Therapeutics Unveils Key Patient-Reported Data for DUVYZAT® in Duchenne Muscular Dystrophy at ISPOR 2025

ITF Therapeutics Presents Vital Patient-Reported Data for DUVYZAT® at ISPOR 2025



ITF Therapeutics LLC, the U.S. branch of the Italian company Italfarmaco, has announced an important poster presentation concerning patient-reported outcome data for DUVYZAT® (givinostat) as it pertains to Duchenne muscular dystrophy (DMD). This presentation is set to take place at the Professional Society for Health Economics and Outcomes Research (ISPOR) conference, occurring from May 13 to 16, 2025, in Montreal, Canada.

The focus of the presentation is on the analyses derived from the double-blind, randomized Phase 3 EPIDYS study. This significant research compares the efficacy and safety of givinostat to a placebo, specifically in ambulant boys aged six and older diagnosed with DMD. Notably, all participants in the study also received corticosteroids, which are often a part of therapeutic regimes for managing DMD.

As a histone deacetylase (HDAC) inhibitor, DUVYZAT has been indicated for treating patients six years and older with DMD. Treatment with this compound aims at mitigating the progression of the progressive muscular dystrophy that characterizes DMD, a condition that severely impacts muscle function and quality of life.

Matt Trudeau, President of ITF Therapeutics, emphasized the necessity of integrating patient and caregiver-reported outcomes into the comprehension of daily living realities for individuals grappling with DMD. According to Trudeau, these reports are crucial as they not only reflect the impacts of the treatment on everyday activities but also enhance the understanding of DUVYZAT's effect on patients’ overall quality of life.

For the evaluation of these patient-reported outcomes, researchers employed the Pediatric Outcome Data Collection Instrument (PODCI). This standardized tool is specifically designed to assess daily activities that significantly influence the quality of life of patients suffering from DMD. The PODCI incorporates a global function score and five distinct subscales—upper extremity function, transfer and basic mobility, sports/physical function, pain/comfort, and happiness. Scores from this tool range from 0 to 100; higher values reflect improved quality of life.

Participants or their caregivers completed the PODCI at the onset of the study and again at 12 and 18 months into the trial. This structured approach allows for a clear comparative understanding of how DUVYZAT potentially affects the various aspects of daily living and overall health of DMD patients.

Poster Presentation Details


  • - Title: Patient-Reported Outcomes From a Phase 3 Study of Givinostat in Patients With Duchenne Muscular Dystrophy
  • - Presenter: Katiana Gruppioni, MPH, Director of Evidence Generation, ITF Therapeutics
  • - Poster Session: 5
  • - Poster Code: PCR230
  • - Presentation Date and Time: Friday, May 16, 2025, from 9:00 to 11:30 a.m. ET
  • - Discussion Period: 9:00 to 10:00 a.m. ET

As for the safety profile of DUVYZAT, there are essential warnings and precautions that healthcare providers should consider. For instance, the drug may induce dose-related thrombocytopenia and other myelosuppression signs. It's advisable to monitor blood counts regularly during the initial treatment period to manage any potential adverse effects effectively.

Furthermore, DUVYZAT has been associated with elevated triglyceride levels, necessitating periodic monitoring, particularly among patients with a history of triglyceride elevation. Gastrointestinal disturbances like diarrhea and abdominal pain are common adverse reactions that may occur, requiring readiness to adjust treatment based on patient response.

About DUVYZAT and ITF Therapeutics


DUVYZAT, developed through lab efforts by Italfarmaco in collaboration with both Telethon and the Duchenne Parent Project in Italy, serves as an FDA-approved option for managing DMD. It acts on overactive HDAC enzymes that contribute to chronic muscle inflammation, impeding muscle repair. The unique ability of DUVYZAT to inhibit these enzymes creates the potential for reduced inflammation and increased muscle repair capacity, thus potentially slowing down muscle loss in DMD patients.

ITF Therapeutics was established in January 2024, focusing on rare disease treatments, with a commitment to reflecting the unique needs of the DMD community. Established as part of Italfarmaco's exemplary track record in the pharmaceutical field since 1938, the company aims to create effective treatment solutions that can enhance the quality of life for individuals affected by rare diseases.

For further details about DUVYZAT and ongoing research initiatives, interested parties can visit www.DUVYZAT.com and www.itftherapeutics.com.

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