Maintaining Our $119 FVE for BioMarin Following Overall Positive Val-Rox Update
BioMarin released initial data from a subset of its phase 3 study of hemophilia A gene therapy val-rox as well as updated (three-year) data from the phase 2 study that appears to confirm the product's efficacy, safety, and potential approval timeline. We're maintaining our $119 fair value estimate. We continue to expect BioMarin to file for approval this year and receive Food and Drug Administration approval and launch val-rox by the end of 2020, setting up long-term sales approaching $2 billion...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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