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

Main article: Fabry's disease

2025: Release of the first batch in Russia in a full cycle

In February 2025, the first batch of Fabagal (agalsidase beta) Russia manufactured in full cycle, including substance, was launched on the market. The project on the localization of the orphan drug - from biosynthesis of the cell line-based substance to the final dosage form - was implemented by the company "" Petrovax Farm together with. NICEM named after N.F. Gamalei This is the first time in history the Russian medicine that the drug for the treatment of a rare disease was completely recreated in ours. to the country Its use will provide therapy to almost twice as many patients due to the lower price.

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Фото: Petrovax Farm

Fabry disease is a rare hereditary disease caused by a deficiency of the enzyme alpha-galactosidase, which leads to the accumulation of lipids in cells and damage to vital organs. Without timely therapy, patients face severe complications, including cardiovascular disease, kidney failure and strokes.

By the age of 40, patients with Fabry disease have disabling complications that can lead to premature death, often as a result of heart attack, stroke or kidney failure. Enzyme replacement therapy is vital for them in order to slow down the progression of the disease and reduce the risk of premature death, - said Evgeny Filippov, MD, professor, chief cardiologist of the Ministry of Health of the Ryazan Region.

Fabagal has been used in world clinical practice for more than 10 years. In Russia, it was registered in 2023. Its launch on the Russian market allowed to reduce the cost of therapy by 40% compared to the price of a drug with a similar active ingredient. Already 50 Russian patients have received therapy with Fabagal since its registration in our country.

In December 2024, Petrovax Farm announced the registration of a drug produced in a full cycle in Russia. The project for the deep localization of the orphan drug, including biosynthesis of the substance, in Russia was completed in 2024 in partnership with the N.F. Gamaleya. The implementation of the project for the production of demanded vital drugs for a full cycle in the country contributes to the achievement of the indicators of the Health Development Strategy in the Russian Federation and ensuring the country's drug safety.

It is essential that patients have continued access to the treatment on which their lives depend. The emergence of an orphan domestic drug, especially its production in a full cycle, is an additional guarantee of the availability of therapy for all Russian patients who need it, - said Yuri Zhulev, co-chairman of the All-Russian Union of Patients.

As of February 2025, only 65% of Russian patients with identified Fabry disease receive treatment. At the same time, according to experts, only 10-15% of people with this disease have been diagnosed as a result of insufficient awareness of specialists and a variety of manifestations of the disease.

Screening in families of patients with a confirmed diagnosis, as well as in patients from risk groups, increases detection. Heart disease ranks first among causes of death in patients with Fabry disease (34% among men and 57% among women).

Therefore, in 2024, the Medical Genetic Scientific Center named after Bochkov together with the company Petrovax Farm has launched a federal cardiac screening program for Fabry disease. Doctors from any region of the country can order free molecular genetic analysis free of charge for their patient if they have risk factors by calling the hotline. This socially significant project is extremely important for the early diagnosis of the disease and the timely start of therapy.

As part of the project, it was possible to test about 300 patients, in 4 of them Fabry's disease was confirmed.

2024: Localization in Russia

On December 13, 2024, Petrovax Farm announced the registration of a local drug for the treatment of Fabri-Fabagal disease (agalsidase beta), produced in a full cycle. This is the first orphan drug in Russia for which the technology of substance production has been transferred. The project was implemented jointly with the N.F. Gamalei.

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Фото: Petrovax Farm

The start of deliveries of locally produced drug is planned in the 1st quarter of 2025. The effectiveness of the technology will allow the production of Fabagal in quantities sufficient to provide therapy to all Russian patients with Fabri disease receiving enzyme replacement therapy with agalsidase beta.

We strive to make available solutions for the treatment of socially significant diseases in Russia. The project for the transfer of Fabagal substance production technology from the cell line was completed as soon as possible - in just two years. We can be sure that all patients will be able to receive high-quality treatment regardless of external circumstances, - said Mikhail Tsyferov, President of Petrovax Farm.

The project to localize the full production cycle of Fabagal in Russia was launched in 2022. In April 2024, Petrovax Farm, together with N.F. Gamalei NITSEM, completed one of the key stages of the project - biosynthesis of the substance using a cell line-based producer. Bringing the drug to market will make therapy for this rare disease more affordable. The cost of Fabagal is already 40% lower than for another drug on the market with a similar INN.

The timely start of treatment allows you to slow down the progression of the disease and prevent the development of severe complications from the side, hearts kidneys, nervous and digestive systems. The earlier Fabry disease is diagnosed, the greater the patient's chances of a long and fulfilling life. Family and selective screening make it possible to identify the disease in the early stages, - said Sergey Valentinovich Moiseev, MD, professor, corresponding member, RAS director of the Clinic named after EM Tareeva, head of the Department of Internal, Professional Diseases and Rheumatology. FSAEI HE "First Moscow State Medical University named after I.M. Sechenov" Ministry of Health Russia

A large-scale project of cardiac screening, which helps to diagnose Fabry disease in a timely manner, is being carried out in Russia in 2024. It was initiated by the Bochkov Medical Genetic Scientific Center together with Petrovax Farm. Thanks to the project, each doctor from any region of the country can order a free molecular genetic analysis for his patient by calling the hotline if he has risk factors. This socially significant project is extremely important for the early diagnosis of the disease, which allows you to start therapy on time.

Fabagal has been used in global clinical practice since 2014. In Russia, it was registered in August 2023 based on the results of clinical studies involving both healthy volunteers and patients with Fabry disease. Before the complete transfer of production technology, the drug was imported from South Korea.

In 2024, 47 patients have already begun therapy with Fabagal. According to Sergei Valentinovich Moiseev, all patients who began receiving therapy at the Clinic named after EM Tareev responded well to the therapy.

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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