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BioMarin Gets Second PKU Approval, Anticipates Slow Ramp-Up For Palynziq

 
• By Joseph Haas

Analysts say black box warning of anaphylaxis risk and REMS were anticipated. Company plans a gradual rollout of Palynziq, focusing first on Phase III clinical trial sites and patients.

Array of blood samples for microscopy and biopsy tissue on blue gradient background

BioMarin Pharmaceutical Inc. is being aggressive on pricing, at least compared to analyst expectations, but realistic about a slow, measured roll-out for Palynziq (pegvaliase), its second approved drug for the rare enzymatic disorder phenylketonuria (PKU). US FDA approved the drug on May 24 and BioMarin announced pricing of $488 per unit, which it anticipates will average out to roughly $192,000 per patient annually.

Analysts generally took a positive view of the approval, even though Palynziq’s label includes a black box warning on the risk of anaphylaxis and a Risk Evaluation and Mitigation Strategy (REMS) that ensures a deliberate launch of the enzyme substitution therapy

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