Two Original Orphan Medicinal Products by GENERIUM Approved for Clinical Studies


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.

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).

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.