EU speeds assessment of Amicus Therapeutics' Fabry monotherapy

First investigational therapy for Fabry disease to be granted Accelerated Assessment. Planned MAA submission on track for 2Q15 to request full approval in European Union

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CRANBURY, N.J.—Amicus Therapeutics, a biopharmaceutical company that specializes in therapies for rare and orphan diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted Accelerated Assessment to the oral small molecule pharmacological chaperone migalastat HCl (migalastat) monotherapy for Fabry patients who have amenable genetic mutations.
John F. Crowley, chairman and CEO of Amicus Therapeutics, Inc., stated, "The designation of Accelerated Assessment in the European Union (EU) demonstrates that the EMA understands the current unmet medical need in Fabry disease as a major public health interest, and with this designation may accelerate the approval and our launch timelines to make migalastat available for patients very rapidly. This is excellent news for so many citizens in the EU and in other geographies around the world. As part of our global strategy, we also plan to submit our new drug application for U.S. approval in the second half of this year. We are committed to getting this personalized medicine approved as quickly as possible for Fabry patients with amenable genetic mutations around the world."
Migalastat is the first investigational Fabry drug to be granted Accelerated Assessment. Amicus requested Accelerated Assessment in accordance with the EMA guidelines, which justify Accelerated Assessment for therapies that are expected to be of major public health interest and therapeutic innovation, addressing the greater unmet needs for maintaining and improving the health of the community. Over the past three years, positive opinions were granted after an Accelerated Assessment for a total of just 14 therapies. The most recent innovative treatment options to have been granted Accelerated Assessment include therapies for rare diseases such as morquio A syndrome, lysosomal acid lipase deficiency  and cystic fibrosis.
Under Accelerated Assessment, the CHMP may shorten the Marketing Authorization Assessment (MAA) review period from 210 days, under standard review, to 150 days under Accelerated Assessment. The CHMP opinion is then reviewed by the European Commission, which generally issues a final decision on EU approval within three months. If approved, Amicus would then begin the country-by-country reimbursement approval process. Amicus is on track to submit the MAA to request full approval for migalastat monotherapy in the EU in the second quarter of 2015.  Amicus previously reported positive Phase 3 data for migalastat in both treatment naive (Study 011, or FACETS) and enzyme replacement therapy switch patients (Study 012, or ATTRACT). Results from these studies have shown that treatment with migalastat has resulted in reductions in disease substrate, stability of kidney function, reduction in cardiac mass, and a positive impact on patient-reported outcomes in patients with amenable mutations.
Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A, referred to here as alpha-Gal). The primary biological function of alpha-Gal is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys and skin. This accumulation of GL-3 is believed to cause the various symptoms of Fabry disease, including pain, kidney failure, and increased risk of heart attack and stroke. It is currently estimated that Fabry disease affects approximately 5,000 to 10,000 people worldwide.
Amicus Therapeutics is a biopharmaceutical company that develops therapies for rare and orphan diseases. The company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. Amicus' lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as next-generation enzyme replacement therapy products for Fabry disease, Pompe disease, and mucopolysaccharidosis I.

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