Although we don't believe in timing the market or panicking over market movements, we do like to keep an eye on big changes -- just in case they're material to our investing thesis.
What: Shares of Raptor Pharmaceuticals (NASDAQ:RPTP), a biopharmaceutical company focused on treating rare, and often fatal, diseases, leapt by as much as 24% after the company received U.S. orphan drug exclusivity for Procysbi from the Food and Drug Administration.
So what: Procysbi, which is a treatment approved by the FDA to treat a metabolic lysosomal storage disorder known as nephropathic cystinosis, will now be protected from any form of competition for seven years, through April 30, 2020. The orphan drug designation is granted to those drugs designed to treat diseases that affect less than 200,000 people. According to Raptor, only an estimated 500 people in the U.S. and 2,000 people worldwide suffer from nephropathic cystinosis.
Now what: While this is clearly great news that Raptor is going to be able to sell Procysbi without any competition for a period of seven years, we also have to understand that it's just a starting point from a sales perspective. The U.S. peak sales estimates for the drug are only $60 million, meaning Raptor is going to need additional drug approvals if it has any hope of turning a profit. With Raptor at a valuation in excess of $500 million, I'm beginning to wonder if the company hasn't exceeded the maximum speculation limit for this flight higher and would certainly suggest taking some profits at these levels.
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Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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