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news.Merck KGaA's biotech division Merck Serono has submitted an application to the European Medicines Agency for the marketing authorization of sapropterin as an oral treatment for significant hyperphenylalaninemia, caused due to phenylketonuria or tetrahydrobiopterin deficiency.
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Data from two international, double-blind, randomized, placebo-controlled Phase III clinical trials in patients with hyperphenylalaninemia due to phenylketonuria show that treatment with sapropterin reduces blood phenylalanine levels and may reduce the need to limit phenylalanine in patients’ diet.
Sapropterin had previously received orphan medicinal product designation for the treatment of hyperphenylalaninemia (HPA), which can cause neurological impairment, from both the European Medicines Agency (EMEA) and the FDA. Sapropterin is being developed in partnership with BioMarin Pharmaceutical.
Roberto Gradnik, head of Merck Serono’s operations for Europe, said: “The filing for sapropterin is an important milestone for patients with phenylketonuria or BH4 deficiency. These are serious, debilitating diseases for which there is currently no drug approved in Europe. This submission underscores Merck Serono’s commitment to address unmet medical needs.”