Press Release

Daiichi Suntory Pharma Entered into License Agreement with BioMarin Pharmaceutical Inc. (USA) for A Congenital Metabolic Abnormalities Agent Biopten (sapropterin hydrochloride)

Daiichi Suntory Pharma Co., Ltd. (DSP; Headquarters: Chiyoda-ku, Tokyo; President: George Nakayama), one of the group companies of Daiichi Pharmaceutical Co., Ltd., and BioMarin Pharmaceutical Inc. (BioMarin; Headquarters: Novato, California, U.S.A.; President: Louis Drapeau) today announced they have entered into a license agreement for DSP’s congenital metabolic abnormalities agent known as Biopten (sapropterin hydrochloride, BH4)

BioMarin has acquired exclusive rights to develop, manufacture and market this agent in the worldwide except Japan. BioMarin plans to start phase II trials of this agent in BH4 responded phenylketonuria patients in the U.S.A.

Reference:
A congenital metabolic abnormalities agent Biopten was developed by Suntory Limited (now Daiichi Suntory Pharma Co., Ltd.) for the treatment of Atypical Hyperphenylalaninemia and launched in 1992 in Japan (marketed by Maruho). Hyperphenylalaninemia is a genetic disease which shows central-nerves system problems such as mental retardation and melanin deficient symptoms by the abnormalities of phenylalanine metabolism.

Hyperphenylalaninemia results from the deficiency of phenylalanine hydroxylase enzyme activity or its cofactor tetrahydrobiopterin, BH4 (Atypical Hyperphenylalaninemia).

Recently, it was reported that new type with phenylalanine hydroxylase deficiency who showed a decrease in blood phenylalanine elevations after BH4 loading and it came to be called BH4 responsive hyperphenylalaninemia.