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BioMarin gets its shot at another blockbuster as FDA OKs rare disease drug pegvaliase

Jean-Jacques Bienaime

BioMarin $BMRN has picked up their latest OK for their rare disease drug portfolio, with the FDA coming through with an approval on pegvaliase, which company execs have pegged as a blockbuster in the making.

In a call with analysts Thursday evening, BioMarin execs noted that they will price the drug at $488 wholesale a unit, with patients needing 1 to 2 units a day. The math sets the average price for 1.5 units at $267,180 as year, with prices ranging up to $356,240, underscoring their commitment to earning more than $1 billion a year from the therapy. The average net including mandatory government pricing, say company reps, should reduce that average to about $192,000.

BioMarin’s shares jumped 5% on the news.

The news was both encouraging and completely expected by analysts, who tend to have a more tempered perspective on the drug’s commercial potential. Leerink’s Joseph Schwartz noted:

Given the black box warning (for anaphylaxis) and complications associated with the initial induction/tiration phase, Palynziq sales are likely to be incremental contributors to BioMarin’s top line in the early yrs. And whereas BioMarin’s long-term goal of >$1B Palynziq sales may be possible, this will require substantial uptake and compliance.

“BioMarin is thrilled to be able to offer this important new therapy to adults with PKU who are unable to control their Phe levels with existing options. The approval of Palynziq is the culmination of more than a decade of perseverance by BioMarin employees dedicated to bringing treatments to PKU adult patients,” said Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin.

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.

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 is intended to eliminate the dietary element, possibly aiming at the most severe cases.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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Catalytic Data Science Charleston, SC
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Flatiron Health New York City or San Francisco

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