Health and Healthcare
Can BioMarin Make Treating Hemophilia More Affordable?
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BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is ready to break above the $1 billion revenue barrier in 2016. This is a feat that marks a major hurdle for many biotech and emerging pharmaceutical companies. Now the company’s stock has gained on hopeful data on its hemophilia treatments. 24/7 Wall St. might caution that this data is very preliminary and that it will be quite some time before it is clear whether BioMarin’s investigational gene therapy treatment for hemophilia A will be a home run.
What really matters here is that hemophilia is one of the conditions that are the most expensive to treat. The average medical costs for hemophilia can be massive for insurers and for people who suffer from it. Hemophilia-Information.com notes that hemophilia therapy is among the most expensive in the world, with a total annual per-patient cost ranging from $60,000 to as much as $1,000,000. The site says:
Many patients are on a prophylactic treatment plan requiring an intravenous(IV) infusion 3 times per week, for life. One infusion for an adult weighing 150 lbs. is approximately $3000 for the medication alone. The average cost for adult patients on a prophylactic regiments at this rate is currently $468,000 annually.
BioMarin said that its BMN 270 showed positive Phase 1/2 clinical data as an investigational gene therapy treatment for hemophilia A. BMN 270 has already received an orphan drug designation from the U.S. Food and Drug Administration (FDA) and from the European Commission. The company noted that Phase 3Â trial design preparation and high volume manufacturing plans are underway.
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