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 the orphan drugmaker Amicus Therapeutics (NASDAQ:FOLD) jumped by over 30% this morning on tremendous volume following a regulatory update for the company's orally delivered Fabry disease treatment migalastat. According to the press release, Amicus met with regulators in the EU and U.S. recently, leading the company to decide to push up its regulatory filing in the EU to the second-quarter of this year, and its U.S. application to the second half of the year.
The biopharma previously planned to submit a marketing authorization application in the EU in the second half of 2015. A U.S. filing, though, was expected to be delayed until the completion of another late-stage trial. The company is seeking an accelerated approval pathway in the U.S. that would allow the drug to be approved while a confirmatory study is under way.
So what: Fabry disease patients are currently limited to treatments that require bi-weekly IV infusions, such as Sanofi's Fabrazyme or Replagal from Shire. Migalastat, by contrast, comes in pill form, giving it a potentially huge competitive advantage over existing therapies.
Although peak sales estimates for the drug only stand at around $200 million, it's important to remember that orphan drugs garner an extended period of market exclusivity, tax breaks, and several other commercial benefits for their manufacturers.
Now what: Orphan drugmakers have been a hot-ticket item in biopharma over the last few years. And with major players like Shire openly stating that they are on the lookout for additional deals in the orphan drug arena, Amicus might start to attract some serious attention as a buyout target following this regulatory update.