A prototype drug for the treatment of spinal muscular atrophy (SMA) has been developed by the Federal Medical and Biological Agency (FMBA). In animal studies, it has shown high efficacy and reduced toxicity compared to its Western counterparts, TASS reports. Registration of the drug is expected in 2029-2030.
Vsevolod Belousov, Corresponding Member of the Russian Academy of Sciences and Director of the Federal Center for Brain and Neurotechnology of the FMBA of Russia, stated at the press conference “Advanced Domestic Developments for the Treatment of Brain Diseases” that the main goal of the development is to create a domestically produced drug, patent-protected, that is at least as effective as international counterparts but offers an improved safety profile.
As Belousov noted, the key achievement was modifying the drug to reduce its impact on the liver and heart, organs that are particularly vulnerable when using current SMA therapies.
“We have managed to create a prototype that, after a single injection, prolongs the life of laboratory mice with SMA from 20 days to almost one year,” the scientist said.
The development benefited from government assistance, with funds allocated by the Russian Federation for comprehensive preclinical and clinical trials. According to experts, the registration certificate for the drug can be obtained by 2029-2030.
Spinal muscular atrophy is a group of inherited diseases that progressively damage and destroy motor neurons. The most common form of SMA is caused by a mutation in the SMN1 gene.
Earlier, in May 2025, the Russian Ministry of Health registered the drug Risdiplam from Promomed. The production is organized at AO Biokhimik. The drug is a generic version of risdiplam and is intended for the treatment of spinal muscular atrophy.
