Amicus Therapeutics recently had its Fabry disease treatment, Galafold, approved by the Australian Therapeutic Goods Administration. As a result of this approval, Amicus Therapeutics will be able to begin dispersing its treatment to those who suffer from Fabry disease and have an amenable mutation. To put that in perspective, nearly 50% of the patients in Australia with Fabry disease also have an amenable mutation.
Obviously, this approval will be incredibly useful to Amicus Therapeutics, as they prepare to increase their distribution. By allowing Fabry disease patients to gain access to Galafold, they will finally have access to a reliable medication that can be taken orally. This is in stark contrast to many other medications for Fabry disease, as Galafold is the first one to be designed for oral consumption.
Although Amicus Therapeutics has gotten initial approval for distribution in the European Union, this approval represents the second time a major market has allowed Galafold to be distributed. Given this news, it’s clear that Amicus Therapeutics will likely be able to expand into other markets as well, allowing them to provide significant relief to Fabry disease patients around the world. Interestingly enough, Amicus Therapeutics is currently looking into several other countries as well. Places like Switzerland and Israel use EU approval as the basis for their own guidelines, which means that Amicus Therapeutics can use the initial approval from them to speed up the review process.
As for Galafold itself, Amicus Therapeutics has designed it to help the body stabilize itself and to break down dysfunctional enzymes (GoogleFinance). Although it is currently reliant on an amenable mutation to be effective, it represents a significant step forward in Fabry disease treatment. In fact, this progress represents the first innovation in over a decade for the disease. Hopefully, Amicus Therapeutics can use this innovation as a stepping stone to an even more effective treatment in the future, although that remains to be seen.
More about Amicus Therapeutics https://www.crunchbase.com/organization/amicus-therapeutics