According to reports published on PhillyPurge.com, FDA allowed Amicus Therapeutics to submit the New Drug Application (NDA) for migalastat. This decision was reached after U.S. FDA and Amicus Therapeutics concluded a series of discussions that gave the latter the leeway to proceed with its NDA for migalastat submission. The New Drug Application is an oral medication for Fabry sickness and was prepared through Subpart H. This was a strategy to accelerate the NDA’s approval process by providing existing data like previous clinical research, and also showing a reduction in the disease-causing substrate (GL-3). Continuous accumulation of the ailment-causing substrate such as kidney failure, stroke, heart disease and pain is the major cause of mortality rate in Fabry disease.
According to Amicus Therapeutics’ Chief Executive Officer and chairman, John F. Crowley, the FDA’s guidance played a significant role of assisting patients living with Fabry complications in the U.S. Furthermore, he said it was a great honor for all those involved in developing migalastat because of what they have achieved so far (https://yourbeautycraze.com/amicus-therapeutics-introduces-galafold-fabry-disease-patients/). The Amicus Therapeutics’ CMO, Jay Barth, said they had provided clinical test data to support migalastat’s approval in Switzerland, Israel, and EU, as well as the ongoing regulatory submissions in Canada, Australian, and Japan.
Clinical Data Provided by Amicus Therapeutics
Amicus Therapeutics performed two major clinical studies to support the drug’s medicinal value on Fabry Disease. According to Jay Berth, the willingness expressed by U.S. FDA to review their migalastat data is a proof that the company’s data is clinically and scientifically based. He expressed optimism that the recent migalastat submissions were evident it would be approved soon.
About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical firm with global operations. It was established in 2002 and became a public entity in 2007 using trading symbol FOLD, which is under NASDAQ (Google Finance). It specializes in developing therapies to assist patients with rare diseases. Some of these rare sicknesses include Fabry, Pompe disease, Epidermolysis Bullosa and genetically connected skin disorder. The firm is headquartered in Cranbury, NJ. Amicus Therapeutics has developed numerous products including migalastat and SD-101, which are in their last stages of development. The company’s objective is to ensure patients with rare diseases have a hope of living longer.