More Breaks for BioMarin

It has been a busy couple of months for Rule Breakers pick BioMarin (Nasdaq: BMRN  ) .

Shares are up more than 30% since the beginning of December after the company won FDA approval to market Kuvan, its enzyme replacement drug. BioMarin launched the drug in the U.S. in early January and says it could bring sales of $35 million to $70 million in 2008.

The wide-ranging forecast is partly because BioMarin doesn't know how many patients will respond to Kuvan, and partly because other factors affect how it's taken -- for example, the weight of patients, which affects the dosage. At the Wachovia health-care conference in Boston today, BioMarin said that both the average weight of patients and the doses they have been taking are tracking higher than expected, which is a positive sign for its sales potential. BioMarin estimates that 50% to 60% of the 13,000 patients in the U.S. with the underlying genetic disorder that Kuvan treats will respond to its use; it's charging $57,000 a year for the drug.

BioMarin filed a marketing application for Kuvan in the European Union in November. The company puts the number of people in the developed world with this genetic disorder at 50,000, and will start a phase 1 trial this quarter for another drug, dubbed Peg-Pal, that treats patients who aren't responding adequately to Kuvan. As with its rapid three-year development timeline for Kuvan, BioMarin plans to have Peg-Pal in phase 3 testing by the middle of 2009, if all goes according to plan.

BioMarin's other lead pipeline candidate, 6R-BH4 for a host of cardiovascular and other disorders, is expected to bring early and mid-stage clinical trial results in the second half of the year and through early 2009. In August, BioMarin also acquired the rights to a preclinical-stage cystic fibrosis drug program and expects to begin human clinical trials for the lead drug within two years.

BioMarin expects to finally become profitable in the second half of 2008, thanks to Kuvan and sales of its other two enzyme-replacement drugs, which are Aldurazyme, marketed by Genzyme (Nasdaq: GENZ  ) , and Naglazyme. Look for full financial guidance when the company announces its fourth-quarter results on Feb. 26.

Read/Post Comments (0) | Recommend This Article (2)

Comments from our Foolish Readers

Help us keep this a respectfully Foolish area! This is a place for our readers to discuss, debate, and learn more about the Foolish investing topic you read about above. Help us keep it clean and safe. If you believe a comment is abusive or otherwise violates our Fool's Rules, please report it via the Report this Comment Report this Comment icon found on every comment.

Be the first one to comment on this article.

Compare Brokers

Fool Disclosure

Sponsored Links

Leaked: Apple's Next Smart Device
(Warning, it may shock you)
The secret is out... experts are predicting 458 million of these types of devices will be sold per year. 1 hyper-growth company stands to rake in maximum profit - and it's NOT Apple. Show me Apple's new smart gizmo!

DocumentId: 566037, ~/Articles/ArticleHandler.aspx, 10/21/2016 10:21:33 PM

Report This Comment

Use this area to report a comment that you believe is in violation of the community guidelines. Our team will review the entry and take any appropriate action.

Sending report...

Today's Market

updated 1 hour ago Sponsored by:
DOW 18,145.71 -16.64 -0.09%
S&P 500 2,141.16 -0.18 -0.01%
NASD 5,257.40 15.57 0.30%

Create My Watchlist

Go to My Watchlist

You don't seem to be following any stocks yet!

Better investing starts with a watchlist. Now you can create a personalized watchlist and get immediate access to the personalized information you need to make successful investing decisions.

Data delayed up to 5 minutes

Related Tickers

10/21/2016 4:00 PM
BMRN $82.28 Down -1.92 -2.28%
BioMarin Pharmaceu… CAPS Rating: ***
GENZ.DL $76.25 Down +0.00 +0.00%
Genzyme Corp CAPS Rating: ****