Posts in Category: Health News

Amicus Therapeutics Develop a New Drug Application for Migasalat

Based in Cranbury New Jersey, Amicus Therapeutics is a biopharmaceutical company committed to the treatment of rare and orphaned diseases. Amicus Therapeutics made its initial public offering in 2007 after a failed round in 2006. The failed 2006 round of the initial public offering would have seen the company take up the AMTX as the trading symbol, but the 2007 one gave them FOLD as the trading symbol. Before this, Amicus Therapeutics had been funded by multiple venture capital firms such as Canaan Partners, Radius Ventures and New Enterprise Associates.


Depending in large part on Chaperone Advanced Replacement Therapy for the development of their medical products, Amicus Therapeutics has majored in the treatment of rare and orphaned diseases especially in the Lysosomal Storage Disorders group (WeeklyOpinion). Other than the Lysosomal Storage Disorders, the company is also committed to the treatment of other diseases such as Fabry and Pompe diseases.


In August 2017, the Amicus Therapeutics announced that it had been granted clearance by the FDA to submit a new drug application (NDA) for their premiere drug, Migasalat. Migasalat, whose official trade name is Galafold, is a chaperone developed specifically for the treatment of Fabry disease. Amicus Therapeutics hopes to launch the drug in the last quarter of 2017, especially after they have submitted the NDA application under subpart H which would grant them faster approval from the FDA. The development of the drug has relied heavily on data that the company has collected from numerous clinical trials and other available data which prove that it certain conditions that the drug causes would lead to a reduction in GL-3. GL-3 is the substrate which causes the Fabry disease. The accumulation of GL-3 in the human system, the data shows, leads to the morbidity of the Fabry disease. Fabry is a potent cause of kidney failure, stroke, pain and heart disease.


A rare and undeniably orphaned disease, the Fabry disease is believed to have affected 3000 people in the United States by current figures. With such small figures compared to other diseases, it is easy for the public to ignore such a disease as inconsequential. Amicus Therapeutics believes that the new drug application will bring reprieve to these individuals and their families.

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