BioMarin to Acquire Global Rights to the Drug Kuvan

Kuvan (sapropterin) is the first treatment approved for hyperphenylalaninemia due to the rare metabolism disorder phenylketonuria (PKU) and has orphan drug exclusivity in Europe until 2020. By returning the rights to BioMarin, Merck is confident that the product will continue to serve the best interest of the medical community and patients.

Pegvaliase-which stands for PEGylated recombinant phenylalanine ammonia lyase-is a Phase III enzyme substitution therapy being developed by BioMarin as a potential therapeutic option for adults with PKU.

BioMarin will now have exclusive global rights to both products, except Kuvan in Japan.

Kuvan (BH4) acts a replacement to this naturally occurring PAH co-factor, in order to keep phenylalanine levels under control.

In addition to Kuvan®, the two companies agreed that Merck will return its option to develop and commercialize Peg-Pal, an investigational drug that is also designed for the treatment of PKU, an autosomal recessive genetic disorder caused by either a defect or a deficiency of the enzyme phenylalanine hydroxylase or its co-factor tetrahydrobiopterin. In addition, Merck Serono will receive €60 million in milestones if combined sales of Kuvan and pegvaliase reach undisclosed cumulative sales thresholds and €125 million for regulatory milestones related to pegvaliase.

Merck in 2005 acquired the rights to Kuvan and Peg-Pal, another PKU treatment, outside the United States and Japan.

“This is a magnificent exchange for BioMarin as it gives various operational and strategic cooperative energies for the Company”, said Chairman and Chief Executive Officer of BioMarin Jean Jacques Bienaime.

Kuvan is approved in 51 countries, including the U.S. “We will influence our set up overall foundation and solid connections inside of the PKU group to guarantee that patients globally have entry to Kuvan, and conceivably pegvaliase upon approbation”.

The sale to the San Rafael, Calif.-based BioMarin ends 10 years of ownership by Darmstadt-based Merck of the Kuvan rights in markets outside the USA and Japan. The full-year total Kuvan revenue to BioMarin is expected in the range of $320 million – $350 million including $70 million – $80 million revenue from new rest of the world territories.