FDA approves Italfarmaco Duchenne drug

March 22, 2024

Italian drugmaker Italfarmaco has received FDA approval for its novel histone deacetylase (HDAC) inhibitor, branded Duvyzat, for Duchenne muscular dystrophy (DMD) in patients aged six years and older.

Duvyzat (givinostat) targets the uncontrolled activity of HDACs in muscles affected by DMD, a disorder resulting from insufficient dystrophin. It aims to suppress the excessive activity of HDAC, tackling the series of reactions that result in muscle harm. This approach helps in mitigating the progression of the disease and decelerates the degradation of muscle tissue.

Following the positive outcome of the EPIDYS trial, one of the largest phase 3 trials for DMD to date, Duvyzat demonstrated a statistically and clinically meaningful improvement in patient mobility and muscle function.

In conjunction with the drug's approval, Italfarmaco has announced the creation of ITF Therapeutics, a new subsidiary based in the U.S., dedicated to advancing treatments for rare diseases and spearheading the commercial launch of Duvyzat in the country. Italfarmaco has also applied for Marketing Authorization in the European Union.

Recent FDA approvals have brought new treatments for DMD. Catalyst Pharmaceuticals' Vamorolone offers a novel approach for patients as young as 2 years old​​. Sarepta Therapeutics' gene therapy, Elevidys, is a first of its kind for children ages 4 to 5, aiming at the genetic cause of DMD​​.  Emflaza, developed Marathon Pharmaceuticals, is approved for patients aged 5 and older, providing a corticosteroid option for all genetic forms of DMD​.