On April 17, the Russian biotechnology company Biocad announced the submission of documents for registration of the country's first original development for gene therapy for hemophilia B. The company's investments in this project will exceed 3 billion rubles — work on the drug has been underway for more than seven years. The international nonproprietary name of the development is arvenacogene sanparvovec. The drug is administered once, which simplifies the treatment process.
The registration dossier is based on data from a clinical study involving more than 20 men with severe hemophilia B. Interim results showed a significant increase in blood clotting factor, which is a key indicator of the drug's effectiveness.
Interim results of the clinical study demonstrated a significant increase in blood clotting factor and its stable efficacy with a good safety profile.
Yulia Linkova, Deputy General Director for Clinical Development and Research at Biocad, noted that the drug will become available to patients within one to three years immediately after receiving a registration certificate. This time is required to complete a number of procedures, such as examination of control samples and connection to various reimbursement channels, which will ensure access to treatment for those who desperately need it.
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