Amicus Therapeutics Fights Fabry Disease

When it comes to fighting diseases, Amicus Therapeutics has made a name for itself given its contribution in the medical field. The American biopharmaceutical company is geographically based in New Jersey. Operating under the official NASDAQ trading name of FOLD, Amicus has been a major pace setter for drug stores in America.

 

The primary focus of Amicus Therapeutics is based on rare as well as orphan diseases. Particularly, there is a focus on disorders like lysosomal storage. The objective of the company is to have a modern platform of production for product development. This is highly based on the generation of Chaperone, an advanced replacement therapy. This platform has a concentration of generated enzyme therapies for replacement.

 

Being a modern facility that initiates modern treatment methods for patients, Amicus Therapeutics registered excellent performance in 2014 when it was noted for having the largest portfolio of molecule pharmacological chaperones. The pharmaceutical industry noted this.

 

As at 2014, Amicus Therapeutics was not as established (Yahoo). There were hardly registered drugs from the company. One of the most established drugs from their store was Galafold, a drug used to treat Fabry disease. It also aims to stabilize mutants like alpha-galactosidase. In partnership with GlaxoSmithKline as well as JCR Pharmaceutical, Amicus Therapeutics investigated co-formulation as well as recombinant with alpha-galactosidase. The partnership lasted three years.

 

New Drug

 

Being focus driven, Amicus Therapeutics recently announced its achievement on the new drug application. Through the drug approval agency, Amicus Therapeutics launched migalastat. This is an oral suspension medication specially designed for a disease called Fabry. According to the terms of launch, the drug will officially be introduced to the industry at the end 2017. Amicus is focused on submitting the formal documents that will allow the management to have a quick approval.

 

Contribution

 

Presently, it is projected that about 3,000 people in America are fighting Fabry diseases (https://www.crunchbase.com/organization/amicus-therapeutics). That is why Amicus Therapeutics is applauded for its input in the industry. The company is inclined towards the belief that migalastat offers treatment. For patients with amenable mutations, Amicus firmly believes that the medicine development will have a positive effect on many lives. Aside from Fabry disease, Amicus is prominent for treating epidermolysis bullos. This is a skin disorder connected to a genetic mutation.

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