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news.Amsterdam Molecular Therapeutics has declared that the European Medicines Agency (EMEA) has approved Orphan Drug Designation to AMT's gene therapy product AMT-021, for the treatment of acute intermittent porphyria (AIP).
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Orphan drug designation for AIP allows AMT to market in Europe for ten years, following marketing approval for AMT-021 from the European Union.
Sander van Deventer, CEO, AMT, said: We are proud to have received this Orphan Drug Designation for the treatment of acute intermittent porphyria.
This designation is an important step in the development of a treatment for this seriously debilitating and potentially lethal disease, he added.
AMT is a human gene therapy company focused on research and clinical development for unmet medical need for therapies of severe (orphan) diseases. The company has expertise in metabolic disorders, liver diseases, blood diseases, disorders of the central and peripheral nervous systems.