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ITF Therapeutics Presents Insightful Research on DUVYZAT™ (givinostat)

ITF Therapeutics LLC, an esteemed branch of the Italfarmaco family, took center stage at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference from March 16-19, 2025, in Dallas, Texas. The company unveiled seven significant abstracts, all focusing on the long-term impact of DUVYZAT™ (givinostat) in the treatment landscape for Duchenne muscular dystrophy (DMD).

This year's conference bore special significance as it marked the one-year anniversary of the FDA's approval of DUVYZAT for DMD in patients six years and older. President of ITF Therapeutics, Matt Trudeau, expressed gratitude for being part of such a pivotal event, emphasizing the company's commitment to sharing new insights derived from ongoing research, particularly from their open-label extension studies involving DMD patients.

Key Data Highlights

Among the standout presentations was a contribution by Dr. Krista Vandenborne from the University of Florida. Her research involved data derived from the Phase 3 EPIDYS study, examining patients treated with givinostat. The findings utilized propensity score matching to align 142 patients treated with givinostat and steroids with a control group of 142 patients receiving steroids alone. One critical observation from this analysis was the median age at which significant loss characteristics in DMD patients appeared. Notably, those treated with givinostat exhibited a median age of 18.1 years for loss of ambulation, a considerable delay compared to the 15.2 years seen in the control group.

In a separate study presented by Dr. Craig M. McDonald from UC Davis Health, the effects of givinostat on pulmonary function were scrutinized. The findings demonstrated that patients who lost the ability to ambulate during follow-up exhibited less decline in their respiratory health when treated with givinostat compared to those on standard steroid therapies.

A fascinating aspect was the poster by Dr. Erika L. Finanger from Oregon Health & Science University, where a clinical trial simulation tool was employed to analyze differences in the progression of forced vital capacity (FVC) among patients. This simulation showcased an enhanced time course for those treated with givinostat, indicating a slower decline in respiratory function.

Moreover, safety profiles remained consistent with existing knowledge, as presented by Dr. John F. Brandsema from the Children’s Hospital of Philadelphia. His analysis assessed the safety of givinostat in patients from an ongoing extension study, showing no new safety concerns and reassuring continuity in the treatment's established safety profile.

Other posters further enriched the conversation, presenting models comparing standard care to givinostat and discussing the design of prospective observational studies aimed at real-world patient outcomes.

About DUVYZAT™ (givinostat)

DUVYZAT stands out as a histone deacetylase (HDAC) inhibitor, specifically approved for DMD treatment in children aged six and above. The innovative mechanism mitigates the overactivity of HDAC enzymes, which can lead to chronic inflammation and reduced muscle repair in DMD patients. Through its approval journey, DUVYZAT has shown promise in enhancing muscle health and promoting repair functionalities, providing hope to countless families grappling with DMD.

Final Thoughts

As ITF Therapeutics continues its mission, Trudeau reiterated their commitment to supporting the DMD community. The presentations made at the MDA Conference not only reflect ongoing research but also symbolize a shared aspiration to empower patients and their families with the tools they need for educated treatment decisions. Fostering partnerships with advocacy groups and healthcare professionals remains a cornerstone for ITF Therapeutics as they progress toward revolutionizing care options for rare diseases.

For more detailed information, please visit DUVYZAT's website and ITF Therapeutics.