Research programme: pharmacological chaperones, enzyme replacements and co-formulations - Amicus Therapeutics

Drug Profile

Research programme: pharmacological chaperones, enzyme replacements and co-formulations - Amicus Therapeutics

Alternative Names: AT 2221; AT3375; ATB 101/Migalastat; Chaperone monotherapy - Amicus Therapeutics; Chaperone-Enzyme Replacement Therapy - Amicus Therapeutics; Chaperone-ERT combinations - Amicus Therapeutics; Duvoglustate/rhGAA; rhGAA/AT-2220; rhGAA/duvoglustat

Latest Information Update: 15 Jan 2018

Price : *
* Final gross price and currency may vary according to local VAT and billing address.
* Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase. A link to download a PDF version of the drug profile will be included in your email receipt. Adis is an information provider. We do not sell or distribute the pharmaceutical compounds written about in this database.

At a glance

  • Originator Amicus Therapeutics
  • Class Alkaloids; Alpha-glucosidases; Glycoside hydrolases; Imino pyranoses; Pharmacological chaperones; Small molecules
  • Mechanism of Action Alpha glucosidase replacements; Glucosylceramidase replacements; Iduronidase replacements; Protein folding stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Glycogen storage disease type II
  • Research Fabry's disease
  • No development reported Alzheimer's disease; Gaucher's disease; Mucopolysaccharidosis I; Parkinson's disease

Most Recent Events

  • 15 Jan 2018 Early research in Fabry's disease (Combination therapy) in USA (IV)
  • 08 Jan 2018 Preclinical trials in Fabry's disease (Combination therapy) in USA (IV)
  • 08 Jan 2018 Amicus Therapeutics plans a clinical trial for ATB 101/Migalastat for Fabrys's disease in 2019
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top