What: After reviewing minutes associated with its discussion with the FDA regarding filing its Fabry disease drug Galafold for U.S. approval, Amicus Therapeutics (NASDAQ:FOLD) will need to push back its previously reported timeline and as a result, its shares are falling by over 30% today.
So what: Previously, Amicus Therapeutics reported that its meeting with the FDA suggested it could file Galafold for approval by the end of this year. However, after reviewing the minutes of their discussion with regulators and communicating with the agency, Amicus Therapeutics is now unsure of when it will be able to file Galafold for approval.
The news is a blow to the clinical stage rare disease drugmaker because the market for Fabry disease treatment is worth hundreds of millions of dollars annually and every month that a filing is delayed is another month without sales in the important U.S. market.
Amicus Therapeutics is considering its alternatives, including getting additional data related to Galafold's impact on the gastrointestinal tract that could support a filing and address safety concerns. The company is also consolidating additional data from prior trials, which will take some time, too.
Because Amicus Therapeutics hasn't decided on the appropriate next step, it no longer anticipates a U.S. filing this year and it isn't certain when a filing could occur.
Now what: Amicus Therapeutics Galafold application has already been filed for approval in Europe and a decision from EU regulators is expected in the first half of 2016.
It's unclear whether or not the FDA's desire for more information suggests the EU will similarly press the pause button, but if EU regulators are satisfied, then Galafold could become available in European markets by the end of next year.
Assuming Amicus gets its ducks in a row and completes a filing in the U.S. in 2016, then it could receive a go-no-go decision on Galafold at some point in 2017. Obviously, winning approval for Galafold in both markets is ideal; however, if Galafold doesn't get approval in the U.S., but does get approval in the EU, then it could still become a top seller for the company.
In 2012, pushback from the FDA led to Shire yanking its application for the Fabry disease drug Replagel and although Replagel is only approved in Europe, its sales are still running at an annualized pace of $468 million exiting the second quarter.
Regardless, this FDA delay is a blow to investors hoping for a quicker path to profitability; especially following the company's recent spending to acquire Scioderm in order to get its hands on the late stage drug Zorblisa, a therapy for a rare pediatric condition.
Overall, a delay in an FDA filing is concerning, but the potential for an EU approval next year, an opportunity to file for approval of Zorblisa by 2017, and a solid balance sheet makes Amicus Therapeutics a company that risk-tolerant investors may want to consider buying on sale.