BioMarin Pharmaceutical Inc. Moves Toward Profitability

BioMarin Pharmaceutical (BMRN -1.53%) released earnings last week with solid revenue growth moving the company toward profitability.

Biomarin Pharmaceutical results: The raw numbers

Data source: BioMarin Pharmaceutical.

What happened with Biomarin Pharmaceutical this quarter?

• Vimizim sales were up 45% year over year, catapulting it to the top of BioMarin's top-selling drugs list. The drug did benefit from some large buys in the Middle East and South Korea, which could reduces sales in later quarters.
• Sales of Kuvan jumped of 19% year over year. The company continues to increase the number of patients in the U.S. taking Kuvan, which grew 8% year over year.
• Naglazyme also benefited from large orders in the Middle East with sales up 25%. A lack of an order from Brazil in the year-ago quarter also helped boost the year-over-year comparison.
• Brineura, a treatment for CLN2, a form of Batten disease, was approved by the Food and Drug Administration and given a positive recommendation by EU regulators.

Image source: Getty Images.

What management had to say

The FDA submission for pegvaliase has been delayed but, as Henry Fuchs, BioMarin's president of worldwide research and development, explained, giving the agency information on the immunologic response to the drug now avoids an inevitable delay later if it was submitted without the extra data:

The agency requested that we provide as much of the immunology data upfront to facilitate their timely completion of the review. So it was a negotiated agreement with the agency about how much to give them in advance and how much to expect them to be undertaking as in process review.

BioMarin got a Rare Pediatric Disease Priority Review Voucher for the approval of Brineura. It sold its first one for $67.5 million a few years ago, so naturally an analyst asked about what the company was going to do with this one. But Jean-Jacques Bienaime, BioMarin's chairman and CEO, played it close to the vest, presumably to not tip off potential buyers about how much value BioMarin places on using the voucher for one of its own drugs:

We haven't really decided at this time. We -- a lot of the products we have in development actually would themselves be getting priority review. We don't know at this time, if pegvaliase will get priority review, so it will be in the equation, but that's not the only thing. So we might look at divesting the voucher but again, we haven't made any final decisions.

Looking forward

Management stuck by its 2017 revenue guidance of $1.25 billion to $1.3 billion, and the company thinks bottom-line earnings will also hit their mark with a profit of $30 million to $70 million on an adjusted basis.

On the pipeline front, the FDA marketing application for pegvaliase should be submitted by the end of the quarter, setting up a potential approval late this year or next year depending on whether the drug gets a priority review. And the next big drug to watch is BMN 270, BioMarin's gene therapy treatment for hemophilia A, which will have additional data at the International Society of Thrombosis and Hemostasis in July.