In a long-term study, new data show that Givinostat continues to delay the progression of Duchenne disease.

Givinostat Duchenne

New data from an ongoing seven-year study by Dr. Paolo Betica, Chief Medical Officer at Italfarmaco,

maker of givinostat, online at the 28th Duchenne International Conference on Pristine Muscular Dystrophy,

held February 20 , was presented.

“We are very encouraged to see that long-term study with Givinostat is still beneficial in boys with DMD

and shows its potential as another treatment,” Betika said in a press release.

This study (NCT03373968) is an extension of the previous Phase 2 trial (NCT01761292)

and is testing the safety and long-term tolerance of givinostat when used with corticosteroids in boys 7 to 11

years of age at the beginning of treatment.

Givinostat is administered as an oral suspension (10 mg / ml) twice daily after meals.

After more than seven years, the average age at which boys treated with givinostat lost the ability to walk was 16 years old.

In comparison, boys who took corticosteroids for at least a year lost the ability to walk at an average age of 13.4.

The decline in respiratory function per year is less among boys taking givinostat than similar patients in previous studies.

A 1.7% to 0% reduction compared to the 4% to 6% annual decline observed in other studies.

Givinostat is intended to improve muscle, reduce inflammation, and reduce fat and fibrosis (ulcers).

It works by blocking enzymes called histone acetone diastylase (HDAC), which limit the accessibility of genes.

Excessive HDACs are thought to be involved in muscle degeneration in DMD and related conditions.

The treatment is also being tested in a phased phase 3 experiment, which is expected to continue until March 2022.

In this experiment, 179 boys aged 6 to 17 years were enrolled.

“Our phase 3 pivotal clinical trial is ongoing and on schedule,” Betika said, adding that “we look forward to

announcing the results in the second quarter of next year.”