Two Original Orphan Medicinal Products by GENERIUM Approved for Clinical Studies

Moscow, Russia, 31 January 2020, GENERIUM, a Russian biopharmaceutical company involved in the development and commercialisation of pharmaceutical products for the treatment of orphan, oncological and socially significant diseases, received an authorization from the Russian healthcare authorities for clinical studies of two original medicinal products used in the treatment of hereditary angioedema and mucopolysaccharidosis type II (Hunter syndrome).

Iduronate-2-sulfatase, capable of penetrating the blood-brain barrier (BBB). The drug is intended for the enzyme replacement therapy of a severe hereditary disease—Hunter syndrome.

Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder in which large sugar molecules called glycosaminoglycans or mucopolysaccharides accumulate in body tissues. Hunter syndrome is caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase. The lack of this enzyme causes heparan sulphate and dermatan sulphate to accumulate in all body tissues, which causes abnormalities in many organs, including the skeleton, heart, respiratory and nervous systems.

The recombinant iduronate-2-sulfatase drugs currently available on the market compensate for the enzyme deficiency in all tissues except for the brain, as iduronate-2-sulfatase is a large protein, which is unable to penetrate through BBB into the brain. GENERIUM has developed a fundamentally new molecule of iduronate-2-sulfatase, having provided it with a capacity to be delivered to the brain neurons as efficiently as to other tissues.

Recombinant C1-esterase inhibitor. The medicinal product is intended to treat hereditary angioedema (HAE).

HAE is a rare disorder associated with the quantitative deficiency or insufficient activity of human C1-inhibitor, which results in uncontrolled internal reactions in the blood. It is manifested as swelling affecting the body. Most commonly, the swelling occurs in the arms, legs, face, abdominal cavity, as well as in the throat. The throat swelling often hinders the air entry into the body, which may cause asphyxia.

Today, the only method of treatment for this disease is the delivery of active C1‑inhibitor into the body. Currently, C1-inhibitor concentrate isolated from the blood plasma of healthy donors or recombinant C1-inhibitor (produced from the milk of genetically modified rabbits) is used. However, these foreign manufactured medicinal products are extremely expensive, which significantly limits their use in the Russian Federation. GENERIUM has developed the first recombinant C1-inhibitor drug that is synthesised in the mammalian cells in vitro, which has reduced the cost of its production and will make it affordable for the HAE patients in Russia and other countries in the future.

Non-clinical studies of these medicinal products in animal models showed their safety and high therapeutic efficacy.

About Generium:

GENERIUM is a full-cycle R&D company, the leader in the Russian pharmaceutical market for orphan diseases. The Company possesses its own R&D park, which is capable of developing any biotech product, from molecule design to commercial production. GENERIUM's manufacturing facilities are organised in strict compliance with the international GMP standards. Our plants produce a unique variety of biotech products and are capable of manufacturing any kind of biomedical cell products.


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