Asubio will use this data to supplement its current filing to the Japanese Ministry of Health, Labour and Welfare for approval of its BH4 product for the treatment of phenylketonuria (PKU) in Japan.
PKU is caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). PAH is required for the metabolism of phenylalanine (Phe), an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures, tremors, and limited cognitive ability.
The application is for a label extension for its current BH4 product marketed in Japan for primary BH4 deficiency. BH4 is the active ingredient in Kuvan.
Jean-Jacques Bienaime, CEO of BioMarin, said: “While Asubio already holds the exclusive rights to its own data to commercialize BH4 for all indications in Japan, this new data greatly expands the clinical data set on treatment of PKU and is expected to accelerate the timing for the label extension of Asubio’s current BH4 product.”
BioMarin will receive a milestone payment for approval as well as double-digit royalties on net sales of BH4 for PKU in Japan.
Kuvan is an investigational oral small molecule for the treatment of PKU being developed in partnership with Merck Serono, a division of Merck KGaA.