FDA approved Palynziq pegvaliase-pqpz from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to treat phenylketonuria (PKU) in patients who have uncontrolled blood phenylalanine levels on current treatment.
The company expects to launch the drug by the end of June. BioMarin spokesperson Debra Charlesworth said the drug will have an annual wholesale acquisition cost (WAC) of $267,000, with an average annual net price of $192,000 after rebates. The label includes a boxed warning for anaphylaxis, and the drug is only available via a REMS.
An MAA for the pegylated recombinant phenylalanine ammonia lyase (PAL) enzyme is under EMA review.
BioMarin already markets the only other approved therapy for PKU, Kuvan sapropterin dihydrochloride, a small molecule formulation of tetrahydrobiopterin (6R-BH4). Sales of that drug were $407.5 million in 2017. BioMarin expects generic competition for Kuvan in 2020.
BioMarin dipped $0.18 to $88.28 on Thursday, but gained $4.20 to $92.48 in after-hours trading after the approval was announced.