The Circle of Kindness Charitable State Foundation, together with the Federal Antimonopoly Service, achieved a significant reduction in the price of the first gene therapy, Elevidys (delandistrogene moxeparvec), produced by the American Sarepta Therapeutics. It is the world’s first single-dose agent for gene replacement therapy in Duchenne muscular dystrophy (DMD) patients. The drug is not registered in Russia.
«As a result of the negotiations, the price of one of the most expensive drugs in the world was reduced by 18%, that is, by €400,000, to €2.2 million», — the press service of the Foundation reported.
Medicines registered in Russia are purchased by the Federal Center for Planning and Organization of Drug Provision of the Ministry of Health at the request of the Foundation. The supplier is determined based on the results of electronic auctions. As for unregistered drugs, the Circle of Kindness acquires them independently after negotiations with manufacturers. For the treatment of Duchenne muscular dystrophy, several expensive medicines are purchased at the expense of the Foundation. However, each of them is suitable only for a certain group of patients with this disease. The reduction in the price of purchased medicines allows the Foundation to continue reviewing new drugs for the treatment of severe and rare diseases for inclusion in the lists.
Elevidys is indicated for children aged 4-5 years with a confirmed mutation in the DMD gene, except for those with deletions in exon 8 and/or exon 9 in the DMD gene. The Foundation noted that clinical trials have shown the most favorable result for children of this age group. The procurement of this drug for them began in 2024. Russia is one of the first countries where the single–dose gene replacement drug became available to children with DMD, and 69 of them have already received the therapy. In total, 531 children with the disease received the assistance of the Foundation in 2021-2024.
Earlier, the co-chairman of the All-Russian Union of Patients, Yuri Zhulev, proposed to increase the age of patients of the Circle of Kindness Foundation to at least 21 years. The discussion surrounding the improvement of drug provision mechanisms for patients over 18 years old was initiated during a meeting of the Committee on Social Policy in February.
The Circle of Kindness Foundation was established in January 2021, following a decree signed by Russian President Vladimir Putin. Its primary mission is to enhance the organization and financial provision of medicines and medical products for children suffering from life-threatening and rare diseases, with a list of 100 conditions covered by the foundation.
