Merck Agrees to Return Kuvan® Rights to BioMarin Pharmaceutical to Strengthen Focus on Core Business - Seite 2

Merck's SPARK study helped pave the way for the treatment of infants
with PKU below 4 years of age with Kuvan(R). 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. A
leading company in the treatment of genetic and rare diseases,
BioMarin is dedicated to improving the treatment options and to
providing all resources needed to continue to make Kuvan(R)
available, as well as to explore potential future therapies in this
area, such as Peg-Pal. Merck will work closely with BioMarin during
the transition to ensure continuous access to Kuvan(R) for patients,
physicians and health authorities.

Kuvan(R) is indicated for the treatment of hyperphenylalaninemia
(HPA) due to PKU in patients of all ages who have shown to be
responsive to Kuvan(R), or due to tetrahydrobiopterin (BH4)
deficiency.

In a conference call later today, (October 1, 2015, 14:00 CEST)
Merck management discusses key focus areas on its healthcare pipeline
in immune-oncology (the call can be followed
live here [http://edge.media-server.com/m/p/5i9jg9t3 ] ).

* Merck Serono also acquired the rights to Kuvan(R) in Canada as a
part of this original agreement in 2005, but returned those rights in
2007. 

About phenylketonuria (PKU)  

PKU is an autosomal recessive genetic disorder caused by a defect
or a deficiency of the enzyme phenylalanine hydroxylase (PAH) or its
cofactor tetrahydrobiopterin (BH4). PAH is required for the
metabolism of phenylalanine, an essential amino acid found in all
protein-containing foods. It affects approximately 1/10,000 newborns
in Europe. If PKU patients are not treated with a
phenylalanine-restricted diet, phenylalanine will accumulate in the
blood and brain to abnormally high levels, thereby resulting in a
variety of complications including mental retardation and brain
damage, mental illness, seizures and tremors, and clinically
significant cognitive problems. Universal systematic newborn
screening programs were developed in the 1960s and early 1970s to
enable diagnosis of all patients with PKU patients at birth.

About Kuvan(R)  

Kuvan(R) (sapropterin dihydrochloride) is the first oral therapy
and approved for the treatment of hyperphenylalaninemia (HPA) due to
phenylketonuria (PKU) in patients of all age who have shown to be
responsive to Kuvan(R) or due to tetrahydrobiopterin (BH4)
deficiency(R). Kuvan(R) was developed jointly by BioMarin
Pharmaceutical Inc. and Merck Serono. Kuvan(R) is to be used in
conjunction with a phenylalanine-restricted diet.

Kuvan(R) is the synthetic form of 6R-BH4, a naturally occurring