Roughly 1,000 boys in Canada are living with Duchenne muscular dystrophyDuchenne muscular dystrophy, and the regulatory clock for givinostat just dropped to 180 days. Health Canada has accepted Italfarmaco’s New Drug Submission for givinostat (Duvyzat) and granted it Priority Review status, a designation reserved for drugs that offer meaningful improvement over existing options. That compressed target, less than two-thirds the standard 300-day benchmark, puts a potential marketing authorization decision before the end of 2026.
The submission rests on the Phase 3 EPIDYS trial, a multicenter, randomized, double-blind, placebo-controlled study enrolling 179 ambulant boys aged six and older across 41 tertiary care sites in 11 countries, with three of those sites in Canada. The trial met its primary endpoint: boys receiving givinostat twice daily on top of corticosteroids showed a statistically significant and clinically meaningful improvement in time to complete a four-stair climb compared with those on corticosteroids and placebo. The most common treatment-related adverse events included decreased platelet count, elevated triglycerides, diarrhea, and abdominal pain, but none of the severe or serious adverse events were treatment-related and none prompted study withdrawal. A long-term open-label extension is tracking these patients for up to eleven years, which is a durability dataset that will matter enormously to Canadian payers when reimbursement negotiations begin.
Givinostat’s mechanism is mutation-agnostic: it inhibits the excessive histone deacetylase activity in DMD muscle cells, independent of which dystrophin gene variant a patient carries. That is clinically significant because DMD is genetically heterogeneous, and many mutation-specific approaches leave large patient subsets ineligible. The drug already holds regulatory approvals in the US, where FDA cleared it in March 2024 for patients six and older, as well as in the UK, EU, and UAE. Canada would extend that footprint to a country where corticosteroids have long served as the backbone of management, often with significant long-term tolerability burdens.
Italfarmaco is establishing a dedicated Canadian affiliate to engage health authorities through the review, which signals that reimbursement strategy is already being shaped in parallel with the regulatory process. The number to watch now is the provincial formulary submission timeline: Priority Review approval and public reimbursement in Canada are separate battles, and the gap between the two has historically delayed real-world patient access by a year or more after a federal nod.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
