The rare Fabry disease treatment Fabagal has received the market approval in Russia

Biotechnological company Petrovax received a market approval^[1][2], for the first biosimilar of agalsidase beta in Russia — Fabagal aimed to treat adults and children from 8 years old with a rare Fabry disease.

Since 2021, Petrovax collaborates with ISU Abxis from South Korea to localize the full-cycle manufacture of Fabagal. By 2024, the synthesis of API and the dosage form manufacturing will be carried out in Russia. Investments will amount to about 2 billion rubles.

The localization project will increase the access to the Fabry disease treatment in Russia and will cover 100% patients’ needs for this vital therapy.

Fabagal will be imported directly to Russia until the technology transfer is completed. In July 2023, inspectors from SID&GP (State Institute of Drugs and Good Practices), a pharmaceutical GMP inspection agency under the Ministry of Industry and Trade of Russia approved the GMP compliance of the ISU Abxis production sites. The first supplies are expected in autumn this year.

The approval was obtained according to the positive clinical studies results observed during the Expert Council «Challenges and perspectives of the Fabry disease diagnostics and therapy» held in April 2023 in Moscow^[3]. Experts considered the crucial role of enzyme replacement therapy for the disease treatment to steady the patient’s condition and reduce some symptoms.

Fabagal is used for long-term enzyme replacement therapy for the Fabry disease, a rare genetic disorder that damages kidneys, causes neuropathic pain in the extremities, congestive heart failure, heart attack and stroke. The disease causes the disability and death of patients if not timely diagnosed and treated as well. Since 2014, Fabagal is approved and used in South Korea.

About Fabry disease

It is a genetic disorder damaging the peripheral and central nervous system, kidneys, heart, digestive and vision systems^[4][5], . According to the Patient Care Organization «Genome», now in Russia there are at least 270 patients, including 24 children with this diagnosis, and only 176 people are treated^[6]. However, some sources estimate the patients amount from 1500 to 5000^[7].

Non-specific symptoms complicate the disease diagnostics, which is manifested at an early stage with neuropathic pain not responding to the pain relief treatment. 76% of men and 64% of women suffer from such pain^[8].

In children, the first manifestation of the disease can start in 2-4 years ^[9] as well as the kidney injury at the age of 3-5 years.

The Fabry disease is caused by lack, absence or malfunction of the enzyme α-galactosidase A that leads to the accumulation of glycosphingolipids (Gb3) in lysosomes of cells^[10]. The disease is a life-threatening and chronic progressive rare pathology, which causes a fatal outcome at the age of 40-50 years if not properly treated^[11].
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¹ Регистрационное удостоверение Фабагал® ЛП-№(002966)-(РГ-RU) https://portal.eaeunion.org/sites/commonprocesses/ru-ru/Pages/CardView.aspx?documentId=6304af8ffb44f110aff5a12b&codeId=P.MM.01, дата доступа 15.08.2023.

² https://grls.minzdrav.gov.ru/GRLS.aspx?RegNumber=&MnnR=&lf=&TradeNmR=Фабагал&OwnerName=&MnfOrg=&MnfOrgCountry=&isfs=0&regtype=1%2c6&pageSize=10&order=Registered&orderType=desc&pageNum=1

³ С. И. Куцев, Румянцев А.Г., Е. С. Васичкина, Н. Д. Вашакмадзе, С. В. Воронин, Е. Ю. Захарова, А. Л. Коротеев, Е. Ю. Красильникова, Моисеев С.В. и др. Резолюция Совета Экспертов «Диагностика и лечение болезни Фабри: перспективы и вызовы» от 20 апреля 2023 года, город Москва. Педиатрия им. Г.Н. Сперанского. 2023; 102 (4): 172–176. https://doi.org/10.24110/0031-403X-2023-102-4-172-176

⁴ Мухин Н.А., Моисеев В.С., Моисеев С.В. и др. Диагностика и лечение болезни Фабри. Клиническая фармакология и терапия. 2013; 22 (2): 11–20.

⁵ Кузенкова Л.М., Намазова-Баранова Л.С, Подклетнова Т.В. и др. Болезнь Фабри: особенности заболевания у детей и подростков. Вопросы современной педиатрии. 2015; 14 (3): 341–348.

⁶ https://orphan-genom.ru/zabolevaniya/bolezn-fabri/istoriya-bolezni-fabri/.

⁷ Журавлева М.В., Химич Т.В., Гагарина Ю.В., Котровский В.А. Клинико-экономический анализ применения ферментозаместительной терапии при болезни Фабри. Клиническая фармакология и терапия. 2019; 29 (4): 83–88.

⁸ Ginsberg L. Nervous system manifestations of Fabry disease: data from FOS – the Fabry Outcome Survey. In: Mehta A, Beck M, Sunder-Plassmann G, eds. Fabry Disease: Perspectives from 5 Years of FOS. Oxford: Oxford PharmaGenesis, 2006. Chapter 23. Available from: https://www.ncbi.nlm.nih.gov/books/NBK11613/.

⁹ Министерство здравоохранения Российской Федерации. Клинические рекомендации. Болезнь Фабри. М., 2019. Доступно по ссылке: https://cr.minzdrav.gov.ru/recomend/318_1.

¹⁰ http://www.rare-diseases.ru/rare-diseases/encyclopediadiseases/1956-fabri/.

¹¹ Министерство здравоохранения Российской Федерации. Клинические рекомендации. Болезнь Фабри. М., 2019. Доступно по ссылке: https://cr.minzdrav.gov.ru/recomend/318_1.