Russia completes first ever transfer of orphan drug substance synthesis technology

NPO Petrovax Pharm LLC (hereinafter referred to as Petrovax) together with Federal State Budgetary Institution The Gamaleya National Center of Epidemiology and Microbiology of the Ministry of Health of the Russian Federation (hereinafter referred to as the Gamaleya NCEM) have completed the substance synthesis technology transfer, a key milestone for the project intended to localize Fabagal® (INN: agalsidase beta, a drug used to treat Fabry disease) production. This was the first substance manufacturing technology transfer ever undertaken within a project aimed at the import substitution of orphan drugs in Russia.

The investment project started in 2022. It involves localizing the full-cycle production of Fabagal®. It took two years for the Gamaleya NCEM and Petrovax to transfer substance synthesis technologies. The Gamaleya NCEM site has already produced the first batches of the agalsidase beta substance. The final stage of the project will be the finished dosage form of the drug produced from the Russian substance at the Petrovax site.

The Russian drug is expected to be launched on the market at the end of this year. The efficiently created technology will enable the company to produce the drug in quantities sufficient to provide therapy to all Russian patients suffering from Fabry disease and receiving enzyme replacement therapy with agalsidase beta.

The project is of great socio-economic importance with its goal to increase the availability of the life-saving treatment for Russian patients. A localized substance synthesis is a key element for import independence and will be able to ensure uninterrupted supplies of the drug to the Russian market.

In 2023, Fabagal® was registered in Russia[1]. The maximum selling price for Fabagal® is 40% lower[2] than that of another drug with the same INN represented on the market. With Fabagal® localized, budget costs will drop significantly by about 500 million rubles per year compared to an alternative therapy (savings could amount to 3.8 million rubles per patient per year). Considering the 5-year time horizon, the amount could exceed 2 billion rubles.

Currently, 176 patients are receiving the therapy[3].

Drug information

Fabagal® (INN: agalsidase beta) is used in adults and children over 8 years of age in enzyme replacement therapy for Fabry disease. This orphan genetic disorder causes kidney damage, neuropathic pain in the extremities, congestive heart failure, heart attack and stroke. If no early diagnosis is made and no proper therapy is chosen, the disease will lead to patient’s permanent disability and death. Enzyme replacement therapy can reduce the level of disability and increase the economically active life expectancy of patients by 5,7 years[4]. Fabagal® has been used since 2014.

1 Fabagal® Marketing Authorization ЛП-№(002966)-(РГ-RU) ®type=1%2c6&pageSize=10&order=Registered&orderType=desc&pageNum=1">Фабагал&OwnerName=&MnfOrg=&MnfOrgCountry=&isfs=0®type=1%2c6&pageSize=10&order=Registered&orderType=desc&pageNum=1

2 State Register of Medicines


4 M.V. Zhuravleva, T.V. Khimich, Yu.V. Gagarina, V.A. Kotrovsky. Clinical and economic impact of screening for Fabry disease in children from high risk groups. DOI: 10.32756/0869-5490-2021-1-36-42
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