BioMarin $BMRN has taken a big step toward a final FDA decision on its latest rare disease drug to reach regulators. The agency accepted the biotech’s application for pegvaliase, handed it a priority review to speed the process, and then likely shoved it back down the line with some special requests on CMC.
Officially, the PDUFA date for the drug is February 28, but the new CMC information demanded by regulators will most likely cause a delay of a few months, says the company, putting a decision back to the end of May.
Pegvaliase is a new drug aimed at phenylketonuria, or PKU. The drug is designed to lower blood phenylalanine (Phe), the key biomarker in play. And that’s just what researchers proved in a Phase III that read out in the spring of 2016.
According to BioMarin, their drug maintained mean blood Phe levels at 527.2 umol/L against a baseline set at 503.9 umol/L. The placebo arm reported an increase over eight weeks from 536 umol/L to 1385.7. That’s good enough for BioMarin’s executive crew to confidently predict blockbuster sales at $1 billion, but a number of analysts have been hedging their bets, lowering their peaks to around $500 million.
BioMarin’s stock didn’t fare well in early 2016, though, because the drug also failed to demonstrate a significant improvement in cognition among patients.
Phe is typically managed by a strict diet, with patients on BioMarin’s Kuvan often able to substantially rein it in. This new injectable, AKA PEG-PAL, is intended to eliminate the dietary element, possibly aiming at the most severe cases.
Leerink’s Joseph Schwartz gives BioMarin good odds — 60% — of an approval, but recognizes that investors have been skittish in light of the failure on neurocognition. The priority review designation, given the track record for follow-up approvals, could be telling.
We believe FDA’s decision to grant priority review could calm investor fears/skepticism as the designation conveys the Agency’s optimism and positive view on the drug’s accrued data thus far. Furthermore, given BioMarin’s experience with Kuvan in PKU, the positive trend may continue for pegvaliase.
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