Amicus Therapeutics Moves a New Drug Therapy Forward


Amicus Therapeutics has developed a new drug that it plans to submit to the U.S. Federal Drug Administration in the fourth quarter of this year. It is an oral medication called “migalastat,” and it will be prescribed to treat Fabry disease. The FDA has been in communication with Amicus Therapeutics and has recently informed the company that it has been approved to submit an NDA for migalastat.


Amicus Therapeutics will submit the drug under Subpart H. This will allow the submission to be accepted for accelerated approval. The NDA will be based on data that Amicus Therapeutics currently has on file, such as the results of the clinical research that has already been performed on the drug.


The studies have shown that progressive accumulation of the disease-causing substrate GL-3 causes the fatality of Fabry disease. Specifically, it leads to the pain, stroke, kidney failure and heart disease associated with Fabry disease. The FDA asked Amicus Therapeutics to complete a Phase 3 study that would have gauged gastrointestinal symptoms, but the agency decided that this is no longer needed.


The Chairman and Chief Executive Officer of Amicus Therapeutics, John F. Crowley, stated that the FDA’s guidance is extremely positive for the many people who have been diagnosed with Fabry disease. He pledged to move the submission process forward quickly and ensures the public that it can receive accelerated approval. He said that everything that has occurred is a testament to the dedication of the patients, physicians and employees who dedicated themselves to creating migalastat.


Amicus Therapeutics’ Chief Medical Officer Jay Barth, M.D. also opined. He stated that he has several clinical studies, and two of them are the most extensive studies of Fabry disease that have ever been done. They have been used to win approval for migalastat in Switzerland, the European Union and Israel. They will also be used to gain approval for migalastat in Australia, Canada and Japan. The CMO is confident that Amicus Therapeutics has potent data to submit to the FDA, and he is expecting it will move them toward approval for migalastat in the United States.

Approximately 3,000 people have been diagnosed with Fabry disease in the United States, so the United States is the most advantageous place for Amicus Therapeutics to treat patients with Fabry disease who have responsive mutations.


The Founder and Executive Director of the Fabry Support & Information Group Jack Johnson stated that he is very glad that Amicus Therapeutics will submit migalastat to the FDA for approval ( He believes that this submission means that we are getting close to helping people obtain an oral therapy that would treat the disease in a way that has never been done before. Mr. Johnson credits Amicus Therapeutics with being an excellent ally for those with Fabry disease for more than 10 years, and he is eager to have migalastat for people with the right mutations.


Migalastat is an oral drug that was designed for people with Fabry disease who have the responsive mutations. People with Fabry disease have a dysfunctional enzyme that can be stabilized with migalastat by clearing up the accumulated disease substrate provided that the patient has the necessary mutations. The necessary mutations are those that have been shown via in vitro assays to be responsive to migalastat. According to Amicus, 35 to 50 percent of Fabry patients all over the world are fortunate enough to have the useful mutations.


Migalastat has received approval by the European Commission, and it was given the name “Galafold.” It is approved as a treatment for Fabry disease for people 16 years of age and up.

What Is Fabry Disease?


Patients with Fabry disease inherited it from their parents (MarketWatch). It is a lysosomal storage disorder. With this disease, alpha-galactosidase A is not present in sufficient numbers, and this interferes with the enzyme’s function of degrading the lipids in the lysosomes. Specifically, the lipids that alpha-Gal A degrades are GL-3 and Gb3. When the alpha-Gal A cannot degrade GL-3, levels of this lipid increase and negatively affect the tissue. Sites where this damage occurs include the kidneys, skin, heart and central nervous system. The disease can cause a patient to suffer from a stroke, kidney failure, heart disease and pain.


Fabry disease is a progressive condition in every patient and can cause damage to the organs of those affected.


About Amicus Therapeutics


The biopharmaceutical company Amicus Therapeutics is based in Cranbury, New Jersey. The types of diseases this company focuses its research on are the lysosomal storage disorders. In particular, the company studies orphan diseases and rare diseases. This company produces medications that are based on Chaperon-Advanced Replacement Therapy and develops enzyme replacement therapies. Amicus Therapeutics has expanded in recent years and now has a laboratory in San Diego.


In 2010, the Michael J. Fox Foundation granted Amicus Therapeutics $500,000 to fund the company’s studies. Amicus used the money in collaboration with the David Geffen School of Medicine at UCLA. The Alzheimer’s Drug Discovery Foundation also gave Amicus Therapeutics a grant worth $210,300 in 2010 for a collaborative effort with Icahn School of Medicine at Mount Sinai at its Alzheimer’s Disease Research Center.


In 2013, Amicus purchased one of its competitors Callidus Biopharma and became the owner of the intellectual property and proprietary materials for the enzyme replacement therapy treatment that is being used to treat Pompe disease.


Currently, Amicus Therapeutics has several therapies for diseases that are not common in the human population. They are also developing further therapies and technologies that will be used to help those with rare and orphan diseases. Along with Fabry disease and Pompe disease, they can also treat Epidermolysis Bullosa. Amicus Therapeutics keeps its eye on people with rare diseases and works to make the lives of people living with these conditions easier.


Amicus Therapeutics’ main drug therapy is migalastat for Fabry disease, but it is also developing a drug that will treat Epidermolysis Bullosa. The therapy is currently being called “SD-101” and is in the late stages of its development.

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