Merck Serono & BioMarin Pharmaceutical (Kuvan Rights) General Information
An oral therapy approved in 51 countries worldwide, including member states of the European Union and the USA. The medication is for the treatment of hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) to reduce blood Phe levels in patients. It is a pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates activity of the residual PAH enzyme to metabolize Phe into tyrosine. It is to be used in conjunction with a Phe-restricted diet.