Russia completes first time ever localization of an orphan drug, including biosynthesis of its drug substance

Petrovax Pharm reports to have completed authorization procedures for the drug Fabagal^® (agalsidase beta) manufactured locally (full-cycle production) and used to treat Fabry disease^[1]. This is the first orphan drug in Russia to have its drug substance synthesis technology transferred in the country. The project has been implemented in cooperation with the N.F. Gamaleya NCEM.

First deliveries of the locally produced drug are scheduled for Q1 2025. This technology is efficient enough to be able to produce Fabagal^® in quantities sufficient to provide therapy for all Russian patients with Fabry disease receiving agalsidase beta enzyme replacement therapy.

The project of Fabagal^® full-cycle production localization in Russia was launched in 2022. In April 2024, Petrovax Pharm, together with the N.F. Gamaleya NCEM, completed one of the key stages of the project—biosynthesis of the drug substance using a cell line-based producer. Bringing the drug to market will make therapy of this rare disease more accessible. The cost of Fabagal is 40 % lower^[2] than that of another drug with the same INN represented on the market.

The large-scale cardiological screening project intended to diagnose Fabry disease in time was initiated in Russia in 2024 by the Bochkov Research Centre for Medical Genetics together with Petrovax Pharm. The project enables every physician from any region of the country to order a free molecular genetic testing for his or her patient with risk factors via the hotline. This socially important project is crucial to ensure the disease is diagnosed early, which means that the therapy can be started as soon as possible.

Fabagal^® has been used in global clinical practice since 2014. It was authorized^[3] in Russia in August 2023 following the clinical studies involving both healthy volunteers and patients with Fabry disease. The drug was previously imported from South Korea before the production technology transfer was fully completed.

This year, 47 patients have already started treatment with Fabagal^®. According to Sergey Moiseev, all patients who started receiving treatment at the E.M. Tareev Clinic responded well to the therapy.

Fabry disease is an inherited progressive orphan disease.

This rare genetic condition presents with a mutation in the GLA gene which disrupts the production of the enzyme α-galactosidase needed to break down complex fats (glycosphingolipids). This results in the accumulation of fats in the lysosomes of cells, triggering a cascade of disorders that result in damage to various organs and systems of the body: heart, blood vessels, kidneys, visual organs, nervous system are affected^[4].

Due to the lack of expert knowledge of the disease and the variety of manifestations of Fabry disease, only 10-15 % of patients with the disease are diagnosed^[5]. Screening in the families of patients with a confirmed diagnosis as well as in patients at risk can increase the chances of identifying the disease. To this end, the Fabry disease cardiac screening project supported by Petrovax Pharma has been launched in 2024.

Familial screening can also detect the disease in children and women who may have insidious Fabry disease. Up to 80 % of patients^[6] complain of neuropathic pain in the hands and feet, predominantly in the palms of the hands and soles of the feet. Gastrointestinal symptoms, decreased sweating and heat intolerance are also common in the early stage of the disease^[7].

Petrovax is a company of novel biopharmaceutical practices with 28 years of successful experience. The product portfolio includes original pharmaceutical products and vaccines, biotechnology drugs and branded generics.

Petrovax is the holder of patents for molecules and pharmaceutical manufacturing technologies in Russia and abroad. The in-house R&D center for pre-clinical studies enables a full cycle of drug development. Petrovax’s modern manufacturing complex in the Moscow Region operates in full compliance with the EAEU and EU GMP standards. The synthesis of APIs, sterile and non-sterile pharmaceutical forms production is performed at the manufacturing complex. The annual production capacity is 160 million doses.