Amicus Therapeutics

3675 Market Street Philadelphia, Pennsylvania United States of America

(215) 921-7600 http://amicusrx.com

Compounds:

  • Migalastat (Galafold)
  • ATB200 / AT2221 (cipaglucosidase alfa / miglustat)

Amicus is passionate about developing medicines to transform the lives of those affected by rare and orphan diseases. The most effective way to do this is through scientifically rigorous, well-designed clinical trials. Clinical trials, grounded in science, are the best way to advance a drug through the regulatory approval process and ultimately to the underserved patients. Our goal is to provide access through marketing authorization.

Expanded Access

Amicus is dedicated to addressing the needs of those individuals who have a serious or potentially life-threatening disease that is under investigation by Amicus and who have exhausted all alternative treatment options, including enrolling in available clinical trials. For these patients, Amicus is committed to careful review of all requests for Expanded Access on an individual, case-by-case basis. Taken into consideration are factors such as a person's current medical condition, based upon information provided by their requesting physician. Refer to the Amicus Expanded Access Policy for further details.

Serious Adverse Events (SAE) should be reported using the SAE Report Form. Please use this form to report Serious Adverse Events within 24 hour of awareness: Safety FAX number: +1-866-422-1278, Email address: SAE Report Form safetyreporting@amicusrx.com

Available Resources