MPS VI is a rare, inherited lysosomal storage disease. It is caused by a genetic defect that results in a deficiency or complete absence of the enzyme N-acetylgalactosamine-4-sulfatase. This enzyme is crucial for breaking down complex sugar molecules called glycosaminoglycans (GAGs, formerly known as mucopolysaccharides), which are essential components of many body tissues.
Without this enzyme, GAGs accumulate in cells, leading to progressive cellular damage. This causes a wide range of severe symptoms, including:
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Growth retardation
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Coarsening of facial features
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Hearing and vision loss
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Skeletal and joint deformities
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Enlargement of the liver and spleen
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Cardiovascular and respiratory failure
Galsulfase is an enzyme replacement therapy drug and represents a recombinant form of the natural human enzyme N-acetylgalactosamine-4-sulfatase.
Upon administration, galsulfase restores sufficient enzymatic activity to break down accumulated mucopolysaccharides and prevent further buildup.
Daniil Talyansky, CEO of GENERIUM JSC, commented:
"This landmark achievement embodies three core principles of our work: adherence to global quality standards, balanced affordability, and efficacy proven through comprehensive research. The approval of our second mucopolysaccharidosis treatment this year underscores GENERIUM's unique expertise in developing complex biological molecules, particularly for lysosomal storage disorders."
In October 2025, GENERIUM received marketing authorization for Clotilia® (verenafusp alfa), an original innovative drug for treating MPS II (Hunter syndrome). Clotilia® features a novel technology designed to deliver the therapeutic enzyme across the blood-brain barrier to address the neurological symptoms of the disease.
Both drugs will be manufactured in Russia on a full-cycle basis at GENERIUM's production facilities.