The filing for approval could mean that Galafold is available as a new therapy for Fabry disease patients as early as next year. If so, Galafold will compete against two of the planet's highest-priced drugs: Sanofi's Fabrazyme and Shire's Replagal. Those drugs cost $200,000 annually and combined had more than $990 million in sales last year.
Fabrazyme and Replagal replace the alpha-galactosidase A (alpha-Gal A) enzyme that is missing in Fabry disease patients, but instead of replacing alpha Gal A, Galafold helps patients who still produce some of the enzyme use it more effectively. Because Galafold has a different mechanism of action than Fabrazyme and Replagal, there's also a potential for its use as an adjunct to these therapies. Studies are ongoing that are evaluating Galafold alongside these drugs and if those studies pan out Galafold could become part of a standard of care for most Fabry disease patients.
Even if those studies fail, however, there could still be a nine-figure market opportunity for Galafold. Amicus Therapeutics estimates that about half of all Fabry disease patients could benefit from taking it as a monotherapy.
Of course, there's no guarantee that the EU regulators will approve Galafold, so investors will need to wait and see what the EU's advisory committee recommends. That committee should offer up its decision before year-end and that could clear the way for an official go-no-go decision in 2016.
Investors also ought to know that Amicus Therapeutics has yet to file for U.S. approval of Galafold, something the company's management expects to do before the end of this year. If Amicus Therapeutics executes on that timeline, then an FDA decision could come by the end of 2016, too.