Maintaining Our BioMarin FVE Following a Steady 1Q and PKU Gene Therapy Announcement
We’re maintaining our $107 fair value estimate for BioMarin following first-quarter 2018 earnings that were in line with our expectations. BioMarin announced that it plans to enter a PKU gene therapy into development in 2019, which we think could be a promising follow-up to the firm’s established presence with oral PKU drug Kuvan and imminent U.S. launch for the more potent injectable PKU therapy pegvaliase. While we expect to see more details on the program later this year, we think the firm’s ...BioMarin Pharmaceutical is a biotechnology company that develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. The company's commercial products are Aldurazyme (laronidase) for Mucopolysaccharidosis I; Brineura (cerliponase alfa) for the treatment of late infantile neuronal ceroid lipofuscinosis type 2; Kuvan (sapropterin dihydrochloride) for the treatment of phenylketonuria; Naglazyme (galsulfase) the treatment of Mucopolysaccharidosis VI ; Palynziq (pegvaliase-pqpz) for adult patients with phenylketonuria; and Vimizim (elosulfase alpha) for the treatment of Mucopolysaccharidosis IV Type A.
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