Amicus Therapeutics Solution For Rare Genetic Disease

Amicus Therapeutics is a global biopharmaceutical organization based out of Cranbury, New Jersey. The company is leading to finding better innovative therapies to treat various rare but severe health conditions.

Amicus Therapeutics leading product migalastat is now available to the patient with Fabry disease in Europe. Galafold (migalastat) is a rare genetic illness characterized by sphingolipids not properly metabolized. Migalastat is an oral-administered medication and the first of its kind to be approved by EMA to treat Fabry illness. The drug is yet to be certified and approved by FDA.

The company has remarkable science, a perfect portfolio and is now focused on finishing their work in the second stage study in Pompe, probably by the end of this year (2017). They have also filed for approval of the leading Migalastat drug in Japan and are planning to do the same here in the US. Other plans for this year include the completion of phase 3 epidermolysis bullosa clinical trial.


One of the agendas of Amicus Therapeutics this year was to introduce its Galafold (Migalastat) treatment for Fabry condition in North America and make plans to see that its FDA approved.

According to the study published in the New England magazine of Medicine, 50 participants with mutant galactosidase alleles were given this medication. However, the study failed to achieve its objectives, and the situation could have been worse if all the 67 participants could have taken part in the research, including patients who could not have benefited from this drug. For FDA to approve this drug in the US, more clinical trials and tests need to be done. Read the company profile at Crunchbase.

The company’s balance sheet is healthy and estimated to be much better by 2024 because of the increase in demand for Fabry’s illness medication.

So if you know someone who is suffering from a rare and devastating condition, including Pompe disease, Epidermolysis Bullosa, and Fabry Illness, Amicus Therapeutics have amazing technologies used to produce innovative drugs to treat patients suffering from the conditions mentioned here. The struggle and pain that these people go through are what motivates Amicus Therapeutics to continue with what they are doing. Their goal is to make these people better and healthy again. Know more on about Amicus Therapeutics.