Italfarmaco has announced exciting Phase 3 results for their investigational drug, Givinostat, for Duchenne muscular dystrophy.

Givinostat acts as an inhibitor, allowing the natural muscle repair process to work more normally. The trial enrolled boys with Duchenne ages six and older who were walking, able to climb stairs, and met other study criteria. People with any genotype could participate, and a total of 179 participants enrolled in the study, including trial sites across Canada.

The drug has shown:

• Preservation of muscle mass
• Delays in disease progression regardless of mutation
• A good safety profile
• The oral suspension was well tolerated

“These topline Phase 3 results in DMD add to the growing dataset we have collected over the last years which have shown positive outcomes with our investigational therapeutic candidate, Givinostat, when taken together with steroids in patients with DMD, providing new hope for boys with DMD, their families and the medical community.

There is a tremendous unmet medical need for additional drugs to treat thisdebilitating rare disease and with these positive results, we intend to meet with regulatory agencies to share these findings and discuss a path forward to submit the complete dataset in a marketing application for potential approval. We are extremely excited to share these new positive analyses from the EPIDYS trial with the community and would like to thank all the stakeholders involved.”

Paolo Bettica, MD, PhD, Chief Medical Officer at the Italfarmaco Group

In October 2020, the U.S. Food and Drug Administration (FDA) granted a Rare Pediatric Disease designation to Givinostat for the treatment of DMD. Earlier, the company also received an Orphan Drug Designation from the FDA and EMA, and a Fast Track designation from the FDA. Based on the study results, which show that the addition of Givinostat to steroid treatment leads to clinical benefits for the individuals in the study, the company plans to meet with US and EU regulators to discuss the potential for marketing application submission and seek input on a future submission of the application for regulatory approval.

There is still much work to be done in Canada and Defeat Duchenne Canada will work with our regulatory agencies and Italfarmaco to advocate for timely access for Canadians.