FDA clears orphan drug Kuvan for PKU

The US Food and Drug Administration has approved Kuvan (sapropterin diHCl), an orphan drug and the first product of its kind cleared to slow the effects of a rare genetic disorder - phenylketonuria - that causes mental retardation, smaller brain size, delayed speech and other neurological problems.

Kuvan was developed by BioMarin Pharmaceuticals of the USA in partnership with Merck Serono, a division of Germany's Merck KGaA. The US firm recently licensed the drug to a Daiichi Sankyo subsidiary for the Japanese market (Marketletter September 24, 2007).