14. Palynziq

BioMarin building
(BioMarin)

Active ingredient: pegvaliase-pqpz
Disease: rare inherited blood disorder phenylketonuria
Peak sales estimate: $500 million
Approved: May 24
Company: BioMarin

The scoop: This is another story of a biotech that saw disappointing results in late-stage testing before managing to nab FDA approval, although it was slapped with a black-box warning for anaphylaxis. The drug, known as Palynziq, is a next-generation rival to BioMarin’s older drug Kuvan, which is licensed for phenylketonuria (PKU) but requires patients to adhere to a restrictive diet. Palynziq, which is designed to break down phenylalanine in the blood, doesn’t require that special diet. But it’s not without its limitations as, given that black-box warning, the FDA is mandating that BioMarin administer a detailed risk-mitigation program, which requires patients to receive their first doses in physicians’ offices before they can give themselves the drug at home. Like several other approvals this year, Palynziq gained an FDA green light after BioMarin published weak data. Back in 2016, the drug hit its primary endpoint in a pivotal phase 3 in PKU, but the study also flubbed a key secondary endpoint for cognitive improvements, with no benefit in inattention or mood scores among the patients, which saw its shares hit. — Ben Adams

14. Palynziq

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