Moscow’s Morozov Children’s City Clinical Hospital will acquire 1312 vials of “Amondys 45” for 288 million rubles, a medication used to treat Duchenne muscular dystrophy, a rare genetic disorder. This drug is not currently registered for use in Russia. The order for the drug supply appeared on the public procurement website on June 5, with applications currently being submitted.

Duchenne muscular dystrophy is a rare disease caused by changes in the dystrophin gene. The condition primarily affects boys, occurring in approximately 1 in every 4,500 male births.

Amondys 45, produced by the American company Sarepta Therapeutics, is designed for patients with a specific dystrophin gene mutation amenable to exon 45 skipping. The clinic requests the supply of 2624 ml of Casimersen (the active ingredient), with each vial containing 2 ml. The medication is administered intravenously once a week.

The disease typically manifests in early childhood, leading to a loss of walking ability in the second decade of life and potentially death in the third. Symptoms commonly appear between the ages of 2 and 4, with impaired motor skills and reduced physical activity. By ages 8 to 12, independent movement is often lost.

Amondys 45 received accelerated approval from the US Food and Drug Administration (FDA) in February 2021, indicating that its therapeutic effectiveness is still under evaluation. Casimersen helps to slow the progression of the disease.

Early diagnosis of Duchenne’s disease is crucial. Without treatment, most patients do not survive beyond their early twenties due to respiratory failure.