BioMarin's Win In Phase 3 Hemophilia Study Makes Its Investment Case Stronger

Terry Chrisomalis
Investing Group Leader

Summary

  • BioMarin achieves primary endpoint of 2-year annualized bleeding rate in phase 3 GENEr8-1 study using the gene therapy valoctocogene roxaparvovec for severe Hemophilia A patients.
  • BLA filing and European reviews of valoctocogene roxaparvovec for severe Hemophilia A patients are expected in 1st half of 2022.
  • The Hemophilia A market is expected to be worth $18.1 billion by 2027. About 50% to 60% of Hemophilia patients are of the severe type.
  • Company guided higher for total 2021 sales for all its drugs and believes that in 2022 it will attain GAAP profitability.
  • I do much more than just articles at Biotech Analysis Central: Members get access to model portfolios, regular updates, a chat room, and more. Learn More »

Female studying DNA samples. Computer screens with DNA sequences

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BioMarin Pharmaceutical (NASDAQ:BMRN) is a good long-term investment to look into. It already has several FDA approvals, but its latest late-stage study met the primary endpoint in patients with severe Hemophilia A. These patients were given

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This article was written by

Terry Chrisomalis
13.46K Followers

Terry Chrisomalis is a private investor in the Biotech sector with years of experience utilizing his Applied Science background to generate long term value from Healthcare.

He is the author of the investing group Biotech Analysis Central which contains a library of 600+ Biotech investing articles, a model portfolio of 10+ small and mid-cap stocks with deep analysis for each, live chat, and a range of analysis and news reports to help Healthcare investors make informed decisions.

Analyst’s Disclosure:I/we have no stock, option or similar derivative position in any of the companies mentioned, and no plans to initiate any such positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Seeking Alpha's Disclosure: Past performance is no guarantee of future results. No recommendation or advice is being given as to whether any investment is suitable for a particular investor. Any views or opinions expressed above may not reflect those of Seeking Alpha as a whole. Seeking Alpha is not a licensed securities dealer, broker or US investment adviser or investment bank. Our analysts are third party authors that include both professional investors and individual investors who may not be licensed or certified by any institute or regulatory body.

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