Deal finally unlocks NHS access to ITF's Duchenne drug

ITF Pharma has reached a commercial arrangement that will make its therapy for Duchenne muscular dystrophy, Duvyzat, an option for more than 500 NHS patients in England.

Final draft guidance from reimbursement authority NICE has recommended oral HDAC inhibitor Duvyzat (givinostat) to treat DMD in ambulatory patients aged six and over; in other words, those who can walk or stand, with or without support.

The drug will be covered immediately by the Innovative Medicines Fund (IMF), according to NICE, while NHS England moves forward with arrangements for routine funding.

The lengthy appraisal – which has extended for almost two years – was delayed while Italfarmaco subsidiary ITF Pharma and NHS England finalised commercial parameters for access and to allow time for the recently changed cost-effectiveness threshold to kick in.

News of the agreement and draft final guidance has caused jubilation for the DMD community, with patient advocacy group Duchenne UK calling it a "landmark day" and a "pivotal moment for people with DMD."

However, the advocacy group's chief executive, Emily Reuben, said: "We are disappointed that this approval is restricted to people with Duchenne who can walk or stand, meaning that anyone who is unable to walk or stand will not be given access under this guidance."

She added that Duchenne UK is "urging the relevant stakeholders to look at all available options to make givinostat available to these patients, who desperately need new treatment options."

There are now two approved treatments for DMD available to patients in England and Scotland, said the charity in a statement, and the decision ends a 'postcode lottery' that has been in place since the Scottish Medicines Consortium (SMC) backed use of the ITF's drug at last December.

In 2024, NICE also backed Santhera's steroid-based Agamree (vamorolone) for all patients aged four and over. Previously, NICE also backed Translarna (ataluren) for ambulatory patients aged two years and older with a nonsense mutation in the dystrophin gene.

Duvyzat is thought to restore muscle regeneration, increase muscle mass, and reduce inflammation and scar tissue accumulation in DMD, a progressive muscle-wasting disease that mostly affects boys and typically leads to a life expectancy of 30 years or less.

It was approved by the MHRA based on data from 120 patients in the placebo-controlled phase 3 EPIDYS study, which showed slower disease progression over 18 months of treatment with Duvyzat, including a slower decline from baseline in the time needed to climb four stairs compared to placebo.