- We sent a package of documents to the Ministry of Health. I hope that in the near future we will start conducting clinical trials," Dmitry Morozov, head of the company's board of directors, told TASS.
He noted that if the tests are successful, then "the probability of import substitution is very high."
The drug has been developed since 2018, and the first experiments began in 2019. An effective dose for the first administration to patients has now been determined. The study wants to include children with SMA under the age of eight months, as well as those with the first manifestations of the disease under the age of six months.
There are three drugs for SMA in the world. All of them are currently registered in Russia. These are Spinraza by the American Biogen, Evrisdi by the Swiss Roche and Zolgensma. "Zolgensma" is taken orally, the first two are administered as injections. The price of "Zolgensma" abroad is more than $ 2.1 million. This medicine is included in the Guinness Book of Records as the most expensive in the world. For Russian children, this medicine helps to buy the Circle of Kindness fund.
In 2020, it became widely known about the enormous problems that exist in Russia with the provision of patients - mainly children - with drugs for the treatment of SMA. Previously, the expenses for the purchase of expensive drugs were borne by the regions, which are often unable to finance such purchases. At the same time, children who were prescribed the drug, but actually never received it, died.