Today, WuXi PharmaTech partner Amicus Therapeutics announced that it plans to submit a new drug application (NDA) to the US FDA in the fourth quarter of 2017, using the oral precision drug migalastat to treat Fabry disease.
Fabry disease is a hereditary lysosomal storage disease caused by a mutation in the GLA gene encoding α-galactosidase A (α-Gal A). The main biological function of α-Gal A is to degrade specific lipids in lysosomes, including globular ceramide (GL-3, or Gb3). Reduced or deleted levels of alpha-GalA activity result in accumulation of GL-3 in affected body tissues, including the central nervous system, heart, kidneys, and skin. Studies have found that progressive accumulation of GL-3 is associated with morbidity and mortality of Fabry disease, leading to a range of symptoms including pain, kidney failure, heart disease and stroke at a young age.
Amicus Therapeutics is one of the world's leading biotechnology companies, working in the treatment of rare diseases. The company has powerful advanced therapies for a wide range of human genetic diseases. Amicus' main developments include the monotherapy small molecule drug partner migalastat for Fabry disease, and small molecule drug candidates for other rare devastating diseases such as Epidermolysis Bullosa disease, Pompe disease.
Migalastat is designed to treat patients with Fabry disease with a susceptible mutation. Based on a proprietary in vitro assay, a susceptible genetic mutation is a mutation that responds to migalastat treatment. The precision medical drug can eliminate the disease-derived substrate (GL-3) accumulated in patients with susceptible mutations by stabilizing the body's own dysfunctional enzyme protein. According to Amicus, approximately 35%-50% of Fabry patients worldwide may have the above-mentioned susceptible genetic mutations. The European Commission (EC) has approved migalastat, a first-line treatment for adults and under 16 years of age under the trade name Galafold®, which has been diagnosed with Fabry disease and has a susceptible mutation. Migalastat has also been approved in countries such as Switzerland and Israel. Japan, Canada and Australia are reviewing their regulatory applications.
Amicus is preparing a new NDA submission under Part H (Subpart H), which stipulates that accelerated approval may be obtained. Amicus intends to effectively reduce the pathogenic substrate (GL-3) data from migalastat and the total data in the completed clinical studies for the NDA submission. Previously, the FDA had asked for clinical trials to assess gastrointestinal (GI) symptoms, and the regulator is no longer asking for this additional three studies.
John F. Crowley, Chairman and CEO of Amicus Therapeutics, said: "This new FDA guidance marks a huge step forward for thousands of American Fabry patients. We are rapidly advancing our NDA submissions and accelerating The approved regulatory pathway for migalastat in the United States. Today is a pioneering moment in the development of migalastat drugs and demonstrates the dedication and perseverance of patients, doctors and researchers working together to develop this precision drug.
Dr. Jay Barth, Chief Medical Officer of Amicus Therapeutics, also said: "Our clinical trial data, including two of the largest key clinical studies that have been completed for Fabry disease, have supported the approval of the drug in the European Union, Israel and Switzerland. And as a basis for our regulatory filings in countries such as Japan, Canada and Australia. The FDA's willingness to review migalastat data reflects the gold standard for treatment development we recognize: science-based, data-driven, patient-centric We believe that the NDA submission has a strong data package, and we look forward to the avenue of migalastat approved in the United States."
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