Merck Serono files sapropterin MAA

Merck Serono, a division of German drug and chemicals firm Merck KGaA, has submitted a Marketing Authorization Application to the European Medicines Agency (EMEA) seeking clearance for use of sapropterin dihydrochloride in the treatment of hyperphenylalaninemia (HPA). Specifically, the MAA requests approval for the drug as a treatment for the condition when associated with either phenylketonuria (PKU) or tetrahydrobiopterin (BH4).

HPA, which is characterized by excessive levels of the amino acid phenylalanine in the bloodstream, is caused by defects in the enzymes which normally process the compound. The disorder can cause significant brain damage in infants and young children, as well as considerable neurological impairment in older sufferers.

Sapropterin, which is being developed in partnership with California, USA-based drugmaker BioMarin Pharmaceutical, is a synthetic version of the co-factor 6R-BH4 that is designed to correct the malfunctioning enzymes responsible for the disease.