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Home Industry News Merck KGaA drug Kuvan gets positive EU opinion

Merck KGaA drug Kuvan gets positive EU opinion

29th September 2008

Merck KGaA has announced it has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency for Kuvan (sapropterin dihydrochloride) as a treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKA) or BH4 deficiency.

The compound had previously received orphan medicinal product designation from the agency for the treatment of HPA.

Merck said BH4 and PKU deficiency are rare diseases caused by genetic defects in the metabolism of phenylalanine – an amino acid.

This defect results in HPA – high levels of the amino acid in the blood – which can cause neurocognitive impairment in adults and serious brain damage in children and infants.

At present, there are around 35,000 patients in the EU with HPA as a result of BH4 or PKU deficiency.

Roberto Gradnik, executive vice-president of commercial Europe at Merck Serono, said: “The efficient therapy of PKU and BH4 deficiency is still an unmet medical need and Merck Serono hopes to be able to provide alternative treatments for these rare diseases.”

He added the availability of Kuvan in Europe will facilitate the improved control of phenylalanine levels by patients while also boosting their quality of life.

Last week, Merck KGaA announced positive results of the Improve study of its new formulation of Rebif (interferon beta-1a) in patients with multiple sclerosis.

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