BioMarin Pharmaceutical (NASDAQ:BMRN) continues to see solid growth from its stable of drugs to treat orphan diseases as it pushes toward its goal for a combined $2 billion in revenue in 2020.

BioMarin Pharmaceutical results: The raw numbers


Q3 2018

Q3 2017

YOY Change


$391.7 million

$334.1 million


Income from operations

($15.7 million)

($13.7 million)


Earnings per share




Data source: BioMarin Pharmaceutical. YOY=year over year.

What happened with BioMarin Pharmaceutical this quarter?

  • Sales of Vimizim jumped 37%, but some of that increase was due to a large order from the Brazilian Ministry of Health.
  • Naglazyme had a similar situation, with a larger order causing sales to jump 43% in the third quarter. By comparison, sales have increased 13% looking at the first three quarters of the year combined, compared with the same period last year.
  • Brineura sales were up a whopping 219%, but since sales were only $3.1 million in the year-ago quarter, the drug, which treats a rare disease called CLN2, still isn't contributing meaningful sales.
  • Sales of Kuvan edged up just 7%, which is understandable as some adult patients switch over to Palynziq, which was approved in May.
  • The launch of Palynziq is going well, with 124 patients paying for the drug at the end of the quarter. Most of those patients transitioned over from clinical trials, but there were 43 patients who hadn't taken Palynziq before. With 68 additional patients in the queue, management felt comfortable guiding for having between 250 and 300 patients on the drug by the end of the year.
Doctor talking to patient in an exam room.

Image source: Getty Images.

What management had to say

BioMarin's chief financial officer, Daniel Spiegelman, highlighted the reasons for the slow launch of Brineura:

We need to be working through reimbursement processes -- that's time consuming. We're utilizing name patient sales approvals in markets where it's appropriate. That's been successful, but time consuming as well. So the combination of those factors are part of -- and the rarity of CLN2 is really the picture of a slow revenue ramp.

While patients switching from Kuvan to Palynziq is bad news in the short term since it cannibalizes sales, it's actually good for BioMarin in the long term, as Jeffrey Robert Ajer, the company's chief commercial officer, explained: "To date, almost 40% of naive patients enrollments have constituted active Kuvan patients. Long term, and in the context of expected loss of exclusivity for Kuvan in two years, this is a very positive dynamic."

Looking forward

Management reaffirmed its 2018 guidance from August for revenue of $1.47 million to $1.53 million. Looking further ahead, the company is shooting for 15% revenue growth for the next two years, which would put revenue at about $2 billion. At that point, vosoritide for achondroplasia and valoctocogene roxaparvovec (val rox), BioMarin's gene therapy for hemophilia, could be approved, accelerating revenue growth.

The company plans to give more information about plans for vosoritide, val rox, and the rest of its pipeline at its R&D day on Nov. 7.

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