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"Acquiring rights to Kuvan in Canada allows BioMarin to better coordinatecommercialization efforts in the North American market," said Stephen Aselage,Senior Vice President, Global Commercial Development of BioMarin. "Thisagreement with Merck Serono comes at an exciting time for the company and thePKU community after Kuvan was approved in the United States last week."
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The terms of the agreement specify a reduction in royalties owed toBioMarin on Merck Serono sales outside the United States and Japan. Based onthe structure of the amended agreement, the reduction in royalties cannotexceed an undisclosed cap.
About Kuvan
Kuvan(TM) (sapropterin dihydrochloride) Tablets is indicated to reduceblood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA)due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). Kuvan isto be used in conjunction with a Phe-restricted diet.
The active ingredient in Kuvan, sapropterin dihydrochloride, is thesynthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzymecofactor that works in conjunction with phenylalanine hydroxylase (PAH) tometabolize Phe. BioMarin and Merck Serono estimate that Kuvan could be apotential treatment option for approximately 30 percent to 50 percent of theestimated 50,000 identified PKU patients in the developed world.
Kuvan has received orphan drug designation from both the U.S. Food andDrug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan hasreceived seven years of market exclusivity in the United States. In November2007, Merck Serono submitted a Marketing Authorization Application (MAA) tothe EMEA for sapropterin dihydrochloride as an oral treatment for patientssuffering from HPA due to PKU or BH4 deficiency. If approved in the EU, itwill receive 10 years of market exclusivity for this indication.
About PKU
PKU, a genetic disorder affecting approximately 50,000 diagnosed patientsin the developed world, is caused by a deficiency of the enzyme phenylalaninehydroxylase. PAH is required for the metabolism of phenylalanine, an essentialamino acid found in most protein-containing foods. If the active enzyme is notpresent in sufficient quantities, Phe accumulates to abnormally high levels inthe blood and becomes toxic to the brain, resulting in a variety ofcomplications including severe mental retardation and brain damage, mentalillness, seizures, tremors, and limited cognitive ability. As a result ofnewborn screening efforts implemented in the 1960s and early 1970s, virtuallyall PKU patients under the age of 40 in developed countries have beendiagnosed at birth. Currently, PKU can only be managed by a Phe-restricteddiet, which is supplemented by nutritional replacement products, like formulasand specially-manufactured foods; however, the strict diet is difficult formost patients to adhere to the extent needed for achieving adequate control ofblood Phe levels. To learn more about PKU, please visit http://www.PKU.com.Information on this website is not incorporated by reference into this pressrelease.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals forserious diseases and medical conditions. The company's product portfoliocomprises three approved products and multiple clinical and preclinicalproduct candidates. Approved products i
