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According to clinical trials conducted in 2024, Geropharm's Ataluren demonstrates complete bioequivalence to the originator formulation. The clinical trial enrolled 100 participants. Russian Ataluren will be manufactured in dosage strengths of 125, 250, and 1000 mg.
Duchenne muscular dystrophy (DMD) is a rare inherited disorder in which the body fails to produce dystrophin, a protein essential for protecting muscle cells from damage. Without it, muscles progressively deteriorate: by age 9-12 years, patients lose ambulatory function; by age 14-16 years, respiratory and cardiac failure develops. Without treatment, median life expectancy is 20-25 years.
Ataluren is a gene therapy agent that can attenuate disease progression in pediatric patients with DMD who have a nonsense mutation. This represents a specific type of genetic defect found in approximately 13% of DMD patients. The drug promotes production of small amounts of dystrophin, which slows muscle degeneration. It does not provide a cure, but can extend a child's independent ambulation for several years.
A Russian study of 24 patients at the National Medical Research Center for Children's Health demonstrated improvement or stabilization in 91% of children under age 7 on the 6-minute walk test, the gold standard assessment for DMD. As the disease progresses, this distance gradually decreases.
Approximately 2,000 DMD patients are registered in Russia; the disease predominantly affects males. For Russian citizens, Ataluren is purchased by the special state fund Krug Dobra, funded by the federal budget. The fund provides therapy for more than 450 children with DMD. The cost of one year of treatment with the originator drug is approximately 30 million rubles.
Source: TASS
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