Amicus Therapeutics has announced that the European Commission has granted full approval for the oral small molecule pharmacological chaperone Galafold™ (migalastat) as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.
Amicus began supplying the market in Germany on Monday 30 May 2016 and will commence the reimbursement processes with healthcare authorities in each of the major European countries.
Galafold is the first oral treatment as well as the first precision medicine for Fabry disease. The broad label includes 269 Fabry causing mutations, which represent 35–50% of all patients with Fabry disease. The label also references a website www.galafoldamenabilitytable.com where EU healthcare providers can quickly and accurately determine which mutations are amenable to Galafold.
“This EU approval for Galafold is a significant advancement in the field of precision genetic medicine and a tremendous milestone for the Fabry community,” said John F Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “This approval also completes our transformation to a global, fully-integrated, commercial biotechnology company focused on rare and devastating diseases. Our world-class commercial and business leadership team has done an outstanding job preparing for this day and is ready for the immediate launch of Galafold in Germany. We will also begin the country-by-country reimbursement processes throughout the EU. We are grateful for the ongoing support from our Amicus employees and the Fabry community, in particular those physicians and patients who participated in the clinical studies of Galafold and their families who made this approval possible. As we move forward with the commercial launch of Galafold, we will continue to invest in the innovation of our pipeline. Our vision today is more focused than ever – to build on our strong science and clinical experience to bring forward the highest quality therapies for Fabry, Pompe, EB and other rare and devastating diseases.”
The EC approval was based on clinical data from two Phase III pivotal studies in both treatment naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT), as well as ongoing long-term extension studies. Fabry disease is a rare genetic disease and potentially life-threatening condition caused by the accumulation of disease substrate (globotriaosylceramide, GL-3) in the lysosome due to a dysfunctional or deficient enzyme. Galafold works by stabilising the body’s own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations. An amenable mutation is one that is responsive to therapy with Galafold based on predefined criteria.
“As principal investigator in both Galafold pivotal studies, I have experience treating both naïve and treatment-experienced Fabry patients with Galafold,” said Derralynn Hughes MA DPhil FRCP FRCPath, Senior Lecturer in Haematology at University College London, UK with clinical responsibilities in haematology and lysosomal storage disorders. “I am pleased that the European Commission has approved this new treatment option and I believe it has the potential to address unmet needs among Fabry patients who have amenable mutations.”
“The EU approval of the first oral precision medicine for Fabry disease is a major step forward for patients in Europe,” said Christine Lavery, President of the Fabry International Network (FIN). “We appreciate Amicus’ commitment to the Fabry community and its dedication to develop high quality therapies for Fabry disease. For the first time in more than a decade, patients with Fabry disease who have amenable mutations now have a choice for an innovative new treatment option.”
François Eyskens, MD, PhD, Department of Experimental Medicine and Pediatrics, University of Antwerp, Antwerpen, stated, “During my 20 years in treating Fabry disease, I am convinced that it is underdiagnosed and that significant unmet need remains among these patients. Galafold is an innovative oral precision medicine with a unique mechanism of action that has demonstrated compelling results in naïve and treatment-experienced Fabry patients who have amenable mutations. I am looking forward to offering a differentiated treatment option for the many Fabry patients who have an amenable mutation.”
The EC approval of Galafold follows the unanimous April 2016 positive opinion granted by the Committee for Medicinal Products for Human Use (CHMP) and applies to all 28 member states of the EU and Liechtenstein, with final authorisation pending in Iceland and Norway. The EC approval provides a platform to begin accessing the more than 70% of the Fabry global market, including the EU member states as well as several international territories that accept the EC approval as the basis for marketing submissions. The Company is also pursuing independent regulatory processes in several international territories outside of Europe, including Japan, Australia and Canada. Amicus also expects to meet with the US Food and Drug Administration (FDA) in the middle of 2016 to clarify a potential US regulatory pathway for Galafold.