A preview of Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company established in 2002 at Cranbury New Jersey. In 2008, Amicus Therapeutics expanded from its single location site in New Jersey to a second research Centre in San Diego. The biotechnology company seeks to treat a range of rare and orphaned disorders. The company product manufacturing systems are anchored on the chaperone advanced replacement therapy (CHART) integrated to enzyme replacement therapies. Amicus Therapeutics enjoys a robust development pipeline program for the treatment of a wide range of human genetic disorders. Additionally, the company had deployed the latest technologies and medicines to cure people with devastating conditions (https://seekingalpha.com/article/4073303-amicus-therapeutics-small-biotech-seems-big-mo). Further, the company envisions to positively impact on the lives of their clients, with the need of patients with rare diseases being the Centre of their incentive science.




Some of the lead products at Amicus Therapeutics include:

  • Migalastat. A lead product targeted to individuals suffering from Fraby disease, based on genetic diagnosis.
  • SD-101. Though in late stage development, the product is set to treat skin blistering and lesions connected with the rare genetic connective tissue disease Epidermolysis Bullosa (EB).
  • ATB200/AT2221 – a paradigm that encompasses enzyme replacement enzymes and pharmacological chaperone to treat Pompe disease.
  • Novel enzyme replacement therapy meant to cure Fabry disease


Ongoing studies:


At Amicus Therapeutics, studies are underway to provide a unique Enzyme Replacement Therapy for Lysosomal Storage Disorders (Crunchbase). Further, a research team is currently working on a Protein replacement therapy for the CDKL5 deficiency. The CDKL5 deficiency is a disorder that manifests itself right from infancy and if not treated may lead to paralysis, visual impairment, scoliosis, sensory impairment and gastrointestinal complications.




Amicus Therapeutics believe in Ethics and compliance in the execution of their daily activities. Further, the company supports programs aimed at fostering patient care with the provision of medical, scientific and educational data to all stakeholders of the company. As part of their Corporate Social Responsibility (CSR), Amicus Therapeutics offers charitable contributions to disadvantaged members of the community and offers educational grants. When undertaking such activities, high priority is given to individuals with or have their beloved ones suffering from CDKL5 deficiency, Epidermolysis Bullosa, and Lysosomal Storage Disorders.


Amicus Therapeutics Leads The Way In Rare And Orphan Disease Research

Amicus Therapeutics is a leading biotechnology company that supports treatment for rare diseases worldwide. The company’s mission is to research and produce pharmaceuticals to treat rare and orphan diseases known as lysosomal storage disorders. Amicus Therapeutics was privately held by venture capitalists until 2007 when the company became an IPO and publicly traded on the NASDAQ. Their company headquarters are in Cranbury, New Jersey.


John Crowley is the CEO of Amicus Therapeutics and has held this position since 2010 (MarketWatch). John Crowley is a well-respected biotechnology entrepreneur, and is the co-founder of Novazyme Pharmaceuticals. John’s leadership skills have helped focus the business on patient driven success. Every employee at Amicus Therapeutics is dedicated to the improvement of those born with rare or orphan diseases. Their business model embraces ethics, compliance and integrity in everything all parts of the company.


Amicus Therapeutics is one of a handful of biotechnology companies who are developing drugs for a small sector of the population who are affected by lysosomal storage disorders. The company has several important drugs in production: Migalastat is a drug formulated to treat patients with Fabry disease. Fabry disease is a rare genetic disorder in which the human body cannot release fat due to a lack of enzymes in the body. A second drug in production is SD-101, a treatment for the genetic tissue disorder called Epidermolysis Bullosa. Amicus Therapeutics focus is on researching the effectiveness of novel enzyme replacement therapies and biologics,which are genetically engineered proteins processed from human genes.


Orphan diseases are classified by the USDA as diseases that affect less than 200,000 people in the United States (GoogleFinance). Due to the nature of rare and orphan diseases research in this sector has been historically underfunded. Amicus Therapeutics received a financial helping hand in 2010 from the Michael J. Fox Foundation in the form of a grant for $500,000. In the same year Amicus Therapeutics was granted $210,300 from the Alzheimer’s Drug Discovery Foundation. Amicus Therapeutics purchased the biotechnology company Callidus Biopharma in 2013, and Scioderm in 2015. The company continues to grow as they work towards important biotechnology therapies.