Petrovax: Fabagal (drug for the treatment of Fabry disease)

2023: Obtaining a Marketing Authorization

The biotechnological company Petrovax has received a marketing ^[1][2] for biosimilar agalsidase beta-orphan preparation Fabagal, which is used for the treatment of Fabry disease in adults and children from 8 years old. The company announced this on August 21, 2021.

Since 2021, Petrovax, in partnership with the South Korean company ISU Abxis, has been implementing an investment project to localize the full production cycle of Fabagal. According to the results of the project, in 2024, the synthesis of the substance and the production of the finished dosage form will be carried out in Russia. Investments will amount to about 2 billion rubles.

The project will make the treatment of Fabry disease more affordable and 100% provide patients with a vital Russian-made drug.

Until the technology transfer is completed, Petrovax will import the Fabagal drug to Russia. Positive conclusions of the Ministry of Industry and Trade of Russia for compliance with GMP requirements of ISU Abxis production sites following the results of the field inspection of FBU "GIDS and NP" were received in July 2023. The first delivery of Fabagal is expected in the fall of 2023.

We strive to provide patients with affordable medicines as quickly as possible. Therefore, we begin to import the finished drug at a price approximately 40% lower than the existing one on the market. At the same time, we are working on a complete technological transfer of Fabagal, including biosynthesis of the substance. The development of engineering batches of the substance starts before the end of the month, and deliveries of a completely domestic drug to the market will be possible in the summer/autumn of 2024, said President of Petrovax Mikhail Tsyferov.

"Fabagal" is used for long-term enzyme replacement therapy of Fabry disease - a rare 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 treatment, the disease leads to disability and death of patients. The drug has been registered and used in South Korea since 2014.

Fabry disease (BF) is an inherited disease characterized by damage to the peripheral and central nervous system, kidneys, heart, digestive system, ^[3], ^[4] According to the ANO Center for Patient Care "Genome," as of August 2023, at least 270 patients were diagnosed with BF in Russia, including 24 children, 176^[5] receive therapy^[6]? according to various literary sources, the real number of patients in Russia can reach from 1500 to ^[7]

The difficulty of identifying and treating such patients lies primarily in the non-specificity of symptoms, they appear in the early stages of neuropathic pain, which cannot be treated with analgesics. It affects 76% of men and 64% of womenGinsberg ^[8]

In children, the first manifestations of the disease can begin at 2-4 ^[9] of ^[10], at about the same age (at 3-5 years old), kidney damage often joins.

The disease occurs against the background of a lack, absence or violation of the functioning of the enzyme α-galactosidase A. This, in turn, leads to the accumulation of glycosphingolipids (Gb3^[11] in the lysosomes of cells. The disease is a life-threatening and chronic progressive rare pathology, in which death without the necessary treatment occurs in 40-50 ^[12]..

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