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Amicus Therapeutics Announces New Drug Application




Amicus Therapeutics has received outstanding news from the U.S. Food and Drug Administration (FDA) that its application for a new drug to treat Fabry disease may go forward. Amicus Therapeutics is expected to submit a new drug application for migalastat sometime during the last quarter of this year. The submission is also being prepared for accelerated approval in hopes that this treatment will make serious headway in combating a debilitating disease, which is a genetic disorder caused by the accumulation of a fat called globotriaosylceramide throughout the body’s cells. Patients who suffer from Fabry disease may experience excruciating pain starting at a very early age as well as many other life-threatening conditions. The most common organs to be affected by Fabry disease are the heart and kidneys. The disease is considered to be fatal in the long-term because of the progressive toll on major organs that accumulate over time. It is difficult for many doctors to diagnose in patients at an early age because its symptoms may be slow to present and are easy to confuse. Genetic testing is available from a doctor.

John Crowley, Chairman, and CEO of Amicus Therapeutics issued a statement on behalf of the company in which he accredited the accelerated submission of migalastat to the hard work and talent of the team at Amicus Therapeutics to ease the suffering of patients with Fabry disease. His statements were echoed by Jay Barth, M.D., who says that he is confident that the data package from clinical trials of migalastat is sufficiently robust to support FDA approval of migalastat on an accelerated basis. This would revolutionize the treatment of this disease because it would be the first oral treatment therapy for Fabry disease on the market.


Prior to the development of migalastat, there were very few options for treatment for patients suffering from Fabry disease, which affects more than 3,000 people in the U.S. The cost of treatment per patient was also about $200,000 because of the rarity of the disease and the complexity of the treatment protocol involved, which meant that many people were unable to afford help without full health insurance coverage. The drug proposed by Amicus Therapeutics presents a significant opportunity for those patients to secure an oral therapy that is expected to be much more affordable and accessible for patients of all backgrounds.


The way that migalastat works are that it stabilizes the dysfunctional enzyme in the body of patients who have amenable genetic mutations. The enzyme can then clear the disease substrate. This is likely possible in about 30 to 50 percent of the patients who suffer from Fabry disease. A patient must respond positively to an in vitro assay before being eligible to take migalastat (GoogleFinance). The assay has been developed by Amicus Therapeutics and would be required of patients before they are prescribed the drug.

The outlook in the regulatory landscape is promising for migalastat because it has already been approved by the European Commission to treat patients aged 16 and older. It is branded under the name Galafold in those countries. There have been multiple regulatory submissions outside of Europe, such as in Australia, Japan, and Canada, which are in varying stages of approval. There have not been any impediments to approval in those countries so far, which leaves patients suffering from Fabry disease around the world very optimistic that a viable treatment plan may soon be available to them. Amicus Therapeutics is currently recruiting patients for its long-term safety study of the drug to assess the safety and efficacy of migalastat.


The fact that Amicus Therapeutics is at the forefront of developing an innovative and effective treatment for this disease should come as no surprise to those who follow the pharmaceutical industry. Ever since Amicus Therapeutics went public in 2007, it has attracted some of the most talented researchers and clinical staff around the world to develop cutting-edge enzyme replacement therapies, which is one of the ways that the company has set itself apart from its competitors in the market. Amicus Therapeutics has recently expanded to include research and development locations in both New Jersey and California.


Amicus Therapeutics has been the recipient of significant research grants, such as a $500,000 award from The Michael J. Fox Foundation in 2010 to support is cooperation with the School of Medicine at UCLA. In addition, Amicus Therapeutics was awarded more than $200,000 in grants from the Alzheimer’s Drug Discovery Foundation for a collaborative study on Alzheimer’s with the Icahn School of Medicine at Mount Sinai. These impressive grants and the company’s ability to recruit some of the top scientists and doctors around the world have allowed it to acquire some of its competitors and expand its reach as one of the most cutting-edge research facilities. Callidus Biopharma was acquired by Amicus Therapeutics in 2013, which allowed Amicus Therapeutics to forge further ahead in enzyme replacement therapy for Pompe disease. Two years later, Amicus Therapeutics acquired Scioderm, which was its largest expansion to date.

Amicus Therapeutics has cemented its role as one of the top pharmaceutical companies focused on “orphan” diseases, which have been defined by the FDA as diseases that affect less than 200,000 people in the U.S. Amicus Therapeutics has been one of the few companies able to capitalize on the incentives offered by the FDA for pharmaceutical companies to pursue clinical trials for drugs to treat these rare diseases and continues to dominate its share of the market. The next rare disease that Amicus Therapeutics has set out to treat is epidermolysis bullosa, which affects up to 40,000 people around the world. It involves issues with connective tissues and results in damage to the skin even after minor injuries. Children who suffer from epidermolysis bullosa are sometimes called “butterfly children” because their skin is so easily cut and bruised. Amicus Therapeutics is also in the second phase of effectiveness trials for its proprietary treatment drug for this condition, which is expected to produce initial data to be shared in sometime this year. This is an exciting development for Amicus Therapeutics because the market share is much larger for this disease than for Fabry disease and has already produced a significant hike in stock prices for the company. Investors have viewed Amicus Therapeutics very favorably in the past quarter and are expected to continue to be bullish on the company with the anticipated announcement of FDA approval of migalastat. Even though Amicus Therapeutics is still a fairly new company in the industry, it is in a strong position to continue to distinguish itself as the market leader for orphan diseases.


More about Amicus Therapeutics at

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