BioMarin Licenses Technology From Leading Cystic Fibrosis Research Laboratory at the University of California, San...(ther this important work. We are pl...)

"We are pleased to add this program to our growing product development pipeline. The cystic fibrosis indication aligns well with our existing product portfolio and core competencies as it is a well-defined and relatively large orphan disease," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "It has a clear clinical and regulatory path and will allow us to leverage our commercial infrastructure targeting specialists."

Cystic fibrosis is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With an incidence of approximately 1/3,500 births, cystic fibrosis is the most common, lethal genetic disease in the U.S. CF affects an estimated 30,000 patients in the U.S. and approximately 40,000 patients outside the U.S. CFTR is a transmembrane protein that functions primarily as a chloride channel in the plasma membrane of epithelial cells. The most common mutation in cystic fibrosis, the deltaF508 mutation, causes the protein to be misfolded and through a cascade of events, leads to mucus buildup and ultimately, organ dysfunction and severe infections in the lungs. The median age of survival of a CF patient is 36.5 years.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 fo
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