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BioMarin Isn't a "Gene Therapy Company"

By Brian Orelli, PhD - May 1, 2019 at 2:29PM

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Despite the biotech's move into gene therapy, its bread and butter are still paying the bills.

BioMarin Pharmaceuticals (BMRN -0.09%) started the year with a slow first quarter coming in under its 15% growth target, but management still feels like the company is in a good position to hit its previously announced 2019 revenue guidance, with sales of Palynziq ramping up and lumpy sales of its other drugs eventually averaging out.

BioMarin results: The raw numbers


Q1 2019

Q1 2018

Year-Over-Year Change


$400.7 million

$373.4 million


Income from operations

($54.0 million)

($44.4 million)


Earnings per share




Data source: BioMarin.

What happened with BioMarin this quarter?

  • Sales of Kuvan, which treats phenylketonuria (PKU), were up 8% year over year.
  • The launch of the company's new PKU treatment for adults, Palynziq, is off to a good start, with 414 patients on drug at the end of Q1, up 60% compared to the end of the previous quarter. Sales are still pretty small at just $12.3 million, but that's to be expected because new patients ramp slowly to the maintenance dose over the first six months or more of treatment.
  • Sales of Brineura were up 77% year over year, but that was off a low base. Revenue came in at $12.2 million as the company makes progress finding patients with CLN2, the neurological genetic disease that Brineura treats.
  • Older drugs Vimizim, Naglazyme, and Aldurazyme contributed year-over-year increases of 7%, 16%, and a year-over-year decrease of 31%, respectively, but revenue from all the drugs fluctuates based on timing of sales, which can be lumpy. Looking at demand, net patient growth for Vimizim was 12%. Naglazyme had 6% patient growth. And sales of Aldurazyme by BioMarin's partner Genzyme increased 20% year over year.
Nurse helping patient with an infusion

Image source: Getty Images.

What management had to say

As BioMarin comes up on three-year data for the midstage clinical trial of valoctocogene roxaparvovec (valrox), its gene therapy for hemophilia, Chief Commercial Officer Jeff Ajer noted:

The most important thing out of the three-year update is the bleeding data. And the two reasons for that are: one, that's the clinical morbidity of a patient's experience, it's what cost the healthcare system a lot of money, it's going to be what payers care a lot about. That's not to say that they won't care about other things, but for sure they're going to care about bleeding rate data. And so, what I think about that is little to no bleeding in the third year is going to be great.

BioMarin plans to file an application to start clinical trials for a second gene therapy, BMN 307 for PKU, but CEO Jean-Jacques Bienaime told investors that it wasn't abandoning the development of other types of therapies that have gotten the company to where it is now, saying, "Although gene therapy is a very important component of BioMarin in the future, at this time, we are not planning on turning into a gene therapy company."

Looking forward

Management reaffirmed its 2019 revenue guidance of $1.68 billion to $1.75 billion. The company thinks it can hit its 15% revenue goal again next year, putting in on track for approximately $2 billion in revenue in 2020. At that point, valrox and vosoritide, which treats a genetic disorder that results in dwarfism called achondroplasia, will hopefully both be approved and can accelerate growth.

An update on the long-term valrox data should be released shortly before the June 7 deadline for submitting the late-breaker abstract to the International Society on Thrombosis and Haemostasis annual meeting. Later in the year, BioMarin plans to make a decision about whether to apply for accelerated approval for valrox using a subset of the data or just wait for the full data for a full approval.

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