Russia’s Geropharm has received a registration certificate for its Duchenne myodystrophy drug Ataluren. It became the first Russian generic of the original Translarna (ataluren) drug, produced by the Irish bioframaceutical company PTC Therapeutics, the Geropharm press service told GxP News.
Duchenne myodystrophy (DMD) is a hereditary, progressive muscle-wasting disease caused by a genetic mutation that prevents the body from producing the protein dystrophin, which is essential for proper muscle function. Insufficient dystrophin leads to progressive muscle weakness, causing a loss of the ability to walk, respiratory muscle failure, and heart failure, which increases the risk of death. DMD is usually first diagnosed when a child is three to four years old, in approximately 1 in 3,500 to 5,000 boys. About 2,000 patients with DMD were registered in Russia as of last year. While there’s no cure for DMD, certain medications may help slow its progression.
Clinical bioequivalence trials of Ataluren with the participation of 100 people have shown its complete identity to the original drug, the press service emphasized. The drug helps the body synthesize dystrophin, slowing down muscle tissue loss, while demonstrating a favorable safety and tolerability profile.
Ataluren will be produced in Russia in a full cycle in dosages of 125, 250 and 1,000 mg. The drug is indicated for ambulatory patients over 2 years of age with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.
The original Translarna is purchased by the Circle of Kindness Foundation. The drug, at 3.5 million rubles (1,000 mg No. 30), ranks 8th among the most expensive medicines in the Russian Federation, according to the Cursor IT company. Earlier this year, another Russian company, Generium, began clinical trials of a gene drug for DMD.