Italfarmaco: Agreement with Georgian Health Ministry ensures access to innovative Duchenne muscular dystrophy treatment

The Italian pharmaceutical company Italfarmaco on Friday released a statement welcoming its newly signed agreement with Georgia’s Ministry of Health, which guarantees the availability of the innovative drug Givinostat for patients suffering from Duchenne muscular dystrophy (DMD).

According to the company, the agreement follows the European Commission’s authorisation of Givinostat in June 2025 and will provide access to the treatment for ambulatory DMD patients aged six years and older who receive the medicine in combination with corticosteroids.

Italfarmaco said the managed access mechanism is designed to ensure that eligible patients in Georgia can receive the innovative therapy and reflects the Georgian Ministry of Health’s ongoing efforts to improve access to treatments for rare diseases.

The company added that it would continue working with the Ministry to support the introduction of the medicine and the development of multidisciplinary care for patients with Duchenne muscular dystrophy across the country.

“This milestone reflects the Georgian Ministry of Health’s strong commitment to improving care for people living with rare diseases and the constructive collaboration between healthcare stakeholders, the Duchenne community and Italfarmaco throughout this process”, Francesco Di Marco, Chief Executive Officer of Italfarmaco Group said.

He added that the company welcomes the agreement and thanks the Ministry for seeking practical solutions that ensure access to treatment for eligible patients.

“We welcome this managed access agreement and thank the Ministry for its dedication to identifying practical solutions for eligible patients. Through our partnership, we look forward to continuing our efforts to support the Duchenne community in Georgia”, Di Marco noted.

According to Italfarmaco, the European Commission’s authorisation was based on results from the Phase III EPIDYS multicentre, randomised, double-blind, placebo-controlled clinical trial involving 179 ambulatory boys aged six years and older.

Participants received either Givinostat or a placebo twice daily in addition to corticosteroid therapy.

The company said the study achieved its primary endpoint, demonstrating a statistically significant improvement in the four-stair climb test.

Positive outcomes were also recorded in secondary endpoints, including the North Star Ambulatory Assessment (NSAA) and MRI-based evaluation of fat infiltration.

According to the company, treatment with Givinostat reduced the deterioration of NSAA scores by 40%, suggesting the medicine has the potential to slow disease progression.

It added that most side effects observed during the trial were mild or moderate. The study findings were published in The Lancet Neurology in March 2024.

Italfarmaco also cited interim findings from the ongoing EPIDYS Open-Label Extension study, which showed that the median age at loss of independent ambulation among patients treated with Givinostat was 17.3 years, compared with 11 to 13.4 years reported in previous studies of patients receiving corticosteroids alone.