In Russia, the transfer of technology for the synthesis of an orphan drug substance has been completed for the first time

Petrovax and the Gamaleya National Research Center for Epidemiology and Microbiology have completed one of the key stages of the project — the synthesis of the substance — to localize the production of Fabagal for the treatment of Fabry disease. This is the first example of the transfer of substance production technology in the project to substituting imports of orphan drugs in Russia.

Russia has completed the first transfer of the technology for the synthesis of an orphan drug substance 10464

The investment project was launched in 2022 and involves the localization of the full-cycle production of the Fabagal drug. For two years, the N.F. Gamaleya National Research Center for Epidemiology and Microbiology and Petrovax carried out the transfer of substance synthesis technologies. To date, the first series of the agalsidase beta substance have been produced at the N.F. Gamaleya National Research Center for Epidemiology and Microbiology. The final stage of the project will be the production of the finished dosage form of the drug from the Russian substance at the Petrovax enterprise.

The Russian drug is expected to be launched on the market at the end of this year. The effectiveness of the technology will make it possible to produce the drug in quantities sufficient to provide therapy to all Russian patients with Fabry disease receiving enzyme replacement therapy with agalsidase beta.

The project is of great socio-economic importance. Its goal is to increase access to life-saving treatment for Russian patients. Localization of the synthesis of the substance is a key element of import independence and can ensure uninterrupted supplies of the drug to the Russian market.

In 2023, the drug Fabagal was registered in Russia1. The maximum selling price for Fabagal is 40% lower2 than for another drug on the market with a similar active ingredient. Localization of the drug Fabagal will significantly reduce budget costs – about 500 million rubles. per year compared to alternative therapy (3.8 million rubles per patient). Taking into account the time horizon of 5 years, this amount could reach more than 2 billion rubles. Currently, 176 patients are receiving therapy.

About the drug

The drug Fabagal (active ingredient: agalsidase beta) is used in adults and children over 8 years of age for enzyme replacement therapy of Fabry disease, an orphan genetically determined disease that causes kidney damage, neuropathic pain in the extremities, congestive heart failure, heart attack and stroke. Without early diagnosis and properly selected therapy, the disease leads to disability and death of patients. Enzyme replacement therapy can reduce the level of disability and increase the economically active life expectancy of patients by 5-7 years4. The drug Fabagal has been used since 2014.


  1. Registration certificate Fabagal® LP-No. (002966)-(RG-RU )®type =1%2c6&pageSize=10&order=Registered&orderType=desc&pageNum=12.
  2. State Register of Medicines %d0%a4%d0%b0%d0%b1%d0%b0%d0%b3%d0%b0%d0%bb&INN=&RegNumber=&OrgName=&Barcode=&OrderNumber=&OuterState =60&PageSize=&OrderBy=pklimprice&OrderType=desc
  4. M.V. Zhuravleva, T.V. Khimich, Yu.V. Gagarin, V.A. Kotrovsky, Clinical and economic rationale for screening for Fabry disease in children at risk, 10.32756/0869-5490-2021-1-36-42