Novartis and the Circle of Kindness Foundation for Children with Severe Illnesses have signed a contract for the supply of Zolgensma. The drug that helps stop the progression of spinal muscular atrophy (SMA) is used once and is considered the most expensive in the world: its cost is about 160 million rubles. As the chairman of the "Circle of Kindness" Alexander Tkachenko told reporters, during the "long and difficult" negotiations, it was possible to achieve "a significant, unprecedented price reduction."

The expert council of the foundation approved the first six applications from children who are shown this drug within the framework of the criteria indicated by the manufacturer: age up to six months or a year, depending on the type of SMA, or no more than a certain period from the moment of manifestation of the disease.

Moreover, for four children, decisions were previously made to refuse due to the uncertainty of the delivery time of the drug, but in anticipation of the conclusion of an agreement with the manufacturer, they were revised.

Children will receive Zolgensma by the end of August.

At the same time, the fund will continue to accept applications from children who may be shown the use of Zolgensma, and will purchase medicine as soon as they are approved.

“Delivery times have been shortened as much as possible. From the moment of the decision of the expert council, we simultaneously submit an application to the supplier company, they begin the procedure for processing documents in the Ministry of Health for the import of an unregistered drug, until this moment we provide blood sampling from the patient in order to send it to Holland and do a test for antibodies to adenovirus. it is used in Zolgensme, - Alexander Tkachenko describes the procedure. - By law, an import permit for an unregistered drug must be issued within five days. In practice, two weeks. We hope that the Ministry of Health will be able to make a decision within the time frame specified by the government decree - five days. ”

At the same time, the application arrives at the plant, and the drug is being manufactured. Manufacturing and delivery of a medicine to Russia takes ten days. “For you to understand the complexity, Zolgensma is delivered at a stable temperature of minus 40 degrees. There are sensors that show how the temperature has changed, whether the drug has deteriorated. Dry ice is added every 48 hours. The drug flies on a separate flight, is opened in a medical facility in the presence of a commission that records data on the temperature regime, and only those doctors who have been trained by Novartis to administer it will be able to use it, ”Tkachenko added.

Earlier, the head of the Circle of Kindness in a video message called on Novartis to speed up the registration of the drug in the Russian Federation: “I do not think it correct that Novartis, selling its drug on the Russian market, does not go through the registration procedure for this drug in Russia.

When the Zolgensma drug is registered, our doctors will have clear, understandable recommendations for its use.

Therefore, I appeal to the management of Novartis with an urgent proposal to expedite the registration of Zolgensma in Russia. "

The chairman of the "Circle of Kindness" also noted that the foundation is looking forward to the introduction of neonatal screening, which will allow detecting SMA in the first days of a child's life, before the first symptoms appear.

In this case, Zolgensma is most effective.

The fund agreed with the manufacturing company that information on the number of such patients will be delivered very quickly, the drug will be quickly sent for manufacture and delivered, thus, it will turn out literally in the first month of the baby's life.

The Circle of Kindness Foundation for the Support of Children with Serious, Life-Threatening and Chronic Diseases, including Rare (Orphan), was established by decree of the President of Russia on January 5, 2021.

In February, the fund decided to include Zolgensma in the list of drugs planned for procurement.

The fund's budget is formed by increasing the personal income tax rate to 15%, which is valid for incomes over 5 million rubles per year. Previously, it was assumed that annually for the fund will be allocated 60 billion rubles, but financing this year, as the head of the "Circle of Kindness" said, will be increased: “The Federal Tax Service has assumed that tax revenues will be more, perhaps 80 billion rubles. All the money will be sent to children, ”Tkachenko said.