BioMarin Pharmaceutical Inc. announced that it has licensed data and intellectual property for the Kuvan (sapropterin dihydrochloride) new drug application (NDA) to Asubio Pharma Co., Ltd., a subsidiary of Daiichi Sankyo.
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.
The application is for a label extension for its current BH4 product marketed in Japan for primary BH4 deficiency. Kuvan is an investigational oral small molecule for the treatment of PKU being developed in partnership with Merck Serono, a division of Merck KGaA, Darmstadt, Germany. BH4 is the active ingredient in kuvan.
"We have had a long-standing relationship with Asubio regarding BH4 as their early work on BH4 was important to our success in Kuvan development. We are pleased to finalize this new agreement to facilitate approval in Japan. 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," said Jean-Jacques Bienaime, chief executive officer, BioMarin.
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 therapeutic for the treatment of PKU. The active ingredient in Kuvan, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe. Clinical data suggest that treatment with Kuvan results in significant reductions in blood Phe levels in BH4-responsive patients. It also may enable some patients to minimize or eliminate highly-restrictive dietary constraints by increasing Phe tolerance levels. BioMarin and Merck Serono estimate that Kuvan could be a potential treatment option for approximately 30 percent to 50 percent of the estimated 50,000 identified PKU patients in the developed world.
Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). If approved, it will receive seven years of market exclusivity in the United States and 10 years in the European Union for this indication. Additionally, the FDA has granted Kuvan Fast Track designation, which is designed to facilitate the development of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.