Amicus Therapeutics Funds Its Ground Breaking New Treatment


Amicus Therapeutics, a publicly traded company, announced their submission of a new drug application, or NDA, to the U.S. Food and Drug Administration. Their application to the FDA was recently permitted by the agency to move forward, and this first stage was completed through written correspondences. The governmental agency officially granted Amicus Therapeutics the right to submit an application for the drug migalastat, a precision medicine that is taken orally. Amicus Therapeutics is listed on the New York Stock Exchange under NASDAQ: FOLD (YahooFinance).

The NDA application will be submitted under the rules for fast approval. This is allowed under Subpart H, which accelerates the process for drug approval under certain conditions. The expedited application will include data collected about the drug’s ability to reduce the presence of GL-3, a substrate linked to Fabry disease. The accumulation of the GL-3 substrate is linked to instances of renal failure, heart disease, stroke, pain, and morbidity. Extreme cases are fatal, and the existing data on migalastat demonstrates a significant reduction in the presence of this substrate.

In addition, the NDA application is not required to include an extra Phase 3 study for gastrointestinal symptoms. This was a previous requirement that has been waived under Subpart H. The application will also include all of the supplementary clinical research data performed on migalastat.


Amicus Therapeutics, Official Statements


The CEO and Chairman of Amicus Therapeutics, John F. Crowley, released some remarks concerning the FDA’s actions on issuing its permission to submit the application for migalastat. He commented that the FDA’s approval would provide substantial benefits for “thousands of people living with Fabry disease in the United States.” He also publicly praised the employees, physicians, and patients who contributed to the development of migalastat as a precision medicine for treating Fabry disease.


The Chief Medical Officer of Amicus Therapeutics, Dr. Jay Barth, also conveyed a high level of confidence in the drug. Dr. Barth mentioned that the existing clinical data supported the case for the approval of migalastat within the European Union, Switzerland, and Israel. Submissions for regulatory approval are also pending in Canada, Japan, and Australia. Amicus Therapeutic company officials expressed confidence about the FDA’s inclination to review the precision drug. Clinical data on migalastat was collected under rigorous conditions that reflect the company’s data-driven approach to conducting science.


The Executive Director of Fabry Support and Informational Group, or FSIG, expressed his confidence in the prospects for approval. Mentioning the planned submission of the NDA for the fourth quarter, Jack Johnson, who founded the group and serves as Executive Director, mentioned the unmet need of patients living with Fabry disease within the United States. The excitement around treating the disease with a new oral medication is shared by doctors and patients. He also cited the active engagement in the Fabry patient community, which spans more than a decade. Patients with “amenable mutations in the U.S.” are expected to benefit from the release of migalastat into the market, according to Mr. Johnson. Current data conducted by Amicus Therapeutics indicate that between 35 to 50 percent of the global population living with Fabry could potentially benefit from this drug. This population is identified as having amenable mutations.

Patient Focus in Therapy


Migalastat is expected to affect more than 3,000 individuals living within the United States who have a positive diagnosis of Fabry disease. This is the largest concentration of the disorder, and the precision drug is expected to reduce the presence of the active substrate that exacerbates the condition. Therefore, this is the geographical region where Amicus Therapeutics believes that the most people can experience a positive outcome from the use of the drug.


Migalastat and Fabry Patients


Migalastat is a precision medication that is prescribed to Fabry patients who demonstrate genetic mutations that are amenable to the treatment. The drug acts on the enzymes responsible for causing the condition, which affects the digestion, heart, and kidneys. Pain is also common in Fabry patients. This disease is caused by the enzyme alpha-galactosidase, or alpha-Gal A, which affects the storage of lysosomes. Mutation of the gene known as GLA causes an enzyme deficiency, which leads to Fabry disease.


The absence or reduction of the alpha-Gal A enzyme within the affected tissues can lead to an excessive amount of the substrate globotriaoslceramide, or GL3, which triggers Fabry disease. The most common symptoms include pain, stroke, kidney failure and heart disease. The onset of Fabry disease can begin in childhood, and it may progress throughout the patient’s lifetime. This medication should not be combined with other enzyme replacement protocols. This medication is also not recommended for Fabry patients who do not have a recognized amenable mutation.


About Amicus Therapeutics


Amicus Therapeutics is a biopharmaceutical company with a global presence. It is based in New Jersey, and it gave its initial public offering in 2007. The NASDAQ symbol for Amicus Therapeutics is FOLD. Previous investors of the company include New Enterprise Associates and Radius Ventures. The company’s signature medicinal product, migalastat, is also known as Galafold, which is classified as a chaperone treatment for patients with treatable forms of Fabry disease. Currently, Amicus Therapeutics contracts the manufacturing of these medications, and it has a second research facility located in San Diego, California. The company has received numerous grants and awards to fund operations during the early stages before the initial public offering was made. This includes grant money from the Michael J. Fox Foundation, Alzheimer’s Drug Discovery Foundation, David Geffen School of Medicine and other groups.


The company develops personalized medicines that can be used in clinical treatment protocols for a variety of diseases. This includes medicines for genetic diseases, connective tissue disorders and enzyme replacement therapies. Specifically, new medicines are being developed to treat Pompe disease, Fabry disease and Lysosomal Storage Disorders, or LSDs. The biologics program develops promising new medicines to treat some of the most difficult disorders ( Amicus Therapeutics particularly specializes in the development of medicines used to treat orphan disorders and other rare diseases. Amicus Therapeutics embraces various emerging technologies and platforms that show the promise of developing medicines for the future.

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