OREANDA-NEWS. The Ministry of Health has registered the drug Zolgensma from pharma manufacturer Novartis in Russia, according to data from the state register of medicines. The company applied for registration last year.
This drug is used for treatment of spinal muscular atrophy (SMA) in children under two years old and is considered the most expensive in the world - one dose costs $2.1 mln (more than 154 mln roubles). The purchase of Zolgensma for children in Russia is handled by the Circle of Kindness Foundation. "Zolgensma is the first gene-based medication for the treatment of SMA, it is administered once.
Spinal muscular atrophy leads to gradual muscle weakening and loss of motor and respiratory functions. In children, the disease occurs about once in every 7,000 newborns and is the most common cause of genetically determined infant deaths. In addition to Zolgensma, Spinraza, which was registered in Russia in 2019, is used to treat the disease. Another treatment for SMA, Risdiplam, was registered last year.
In May, the Russian biotechnology company Biocad announced the completion of early development of the first domestic drug for SMA. The drug, called ANB-4, has tentatively shown efficacy in animals with SMA, and the company must now confirm these results in pre-clinical and clinical trials.
Olga Germanenko, head of the SMA Family Foundation, told the Novaya Gazeta newspaper that it usually takes about two to six months from the time a drug is registered until it enters the civilian market. According to her, during this period the availability of the drug decreases, because it has to be imported in Russian packaging, and it takes time to create it and draw up instructions in accordance with the Russian labeling. "During this period it is not possible to import the drug, as it was done before registration, in international packaging,"- she explained.
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