Italfarmaco S.p.A. has announced that the full results from the pivotal phase 3 EPIDYS clinical trial of givinostat in ambulant Duchenne patients six years of age and older have been published in The Lancet Neurology. Givinostat is Italfarmaco’s proprietary histone deacetylase (HDAC) inhibitor for the potential treatment of Duchenne. The EPIDYS clinical trial met its primary endpoint of change in four-stair climb assessment from baseline to 72 weeks.

Givinostat-treated patients also showed favorable outcomes on key secondary endpoints assessed in the study. Taken together, these data suggest the potential of givinostat to delay disease progression when added to corticosteroid treatment. 

In November 2023, Italfarmaco announced an extension in the FDA’s review of the New Drug Application (NDA) for givinostat to allow additional time to review information submitted by Italfarmaco as part of the NDA process. FDA extended the Prescription Drug User Fee Act (PDUFA) date, which is the deadline set by the FDA for their decision about whether to approve an investigational product, to March 21, 2024. 

PPMD is pleased to hear this topline data and we are optimistic that the FDA is considering these positive results as part of a thorough review of Italfarmaco’s NDA for givinostat. We eagerly await news regarding the PDUFA date this Thursday, March 21st and are hopeful that givinostat will be another approved therapeutic option for the Duchenne community. 

ITF Therapeutics, LLC in the U.S.

Italfarmaco also shared information about the launch of a new division of the company in the United States called ITF Therapeutics, LLC that is dedicated to making therapeutic candidates for rare diseases available to patients. Italfarmaco indicated that the company will be providing more information about plans to ensure that givinostat can be accessed by appropriate patients in the U.S. if approved by sharing updates via the new ITF Therapeutics website, which will be available in the coming days. PPMD is pleased to learn about ITF Therapeutics and we look forward to further updates from the company leading up to and following the givinostat PDUFA date.

Read Italfarmaco’s community update about ITF Therapeutics:

Italfarmaco S.p.A. Launches ITF Therapeutics, LLC to Bring New Product Candidates for Rare Diseases to Patients in the U.S.

In January 2024, the private global pharmaceutical company Italfarmaco S.p.A. (headquartered in Milan, Italy) launched a new division in the United States called ITF Therapeutics, LLC that is dedicated to making therapeutic candidates for rare diseases available to patients. I am very pleased to share with you some exciting details about this new division and our goal to make a positive difference for people living with rare diseases.

ITF Therapeutics is led by a team of industry specialists who have spent their careers working to bring innovation to patients, caregivers, providers, and communities affected by rare diseases. We understand and value the critical role that rare disease advocates play in sharing insight, guiding research, championing patient interests, building awareness, and shaping policy. In fact, support from patient advocacy organizations was essential in helping Italfarmaco research and develop our product candidate, givinostat, being evaluated as a potential therapeutic option for Duchenne muscular dystrophy (DMD).

As we await FDA final review of givinostat, we are building the platform to ensure that this potential therapeutic option can be accessed by appropriate patients in the U.S. if approved. We strive to put the patient voice at the center of our mission and will work tirelessly to support the DMD community and deliver more innovation for rare diseases. For more information about our plans, we will be sharing updates via our new ITF Therapeutics website that will be live in the coming days.