Amicus Therapeutics

3675 Market Street Philadelphia, Pennsylvania United States of America

Compounds:

Migalastat (Galafold)
Migalastat (Galafold)

Migalastat HCl is currently licensed in the United States, Canada, Europe, Switzerland, Israel and other countries for the long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease with a mutation amenable to this treatment. The market authorization process is ongoing in multiple other countries.

Migalastat therapy is dosed as one capsule, 123 mg, every other day. Capsules are blister-packaged with an inactive reminder day to assist compliance.

Amicus will receive requests for access prior to market authoziation, through Early Access Care, for individuals meeting the following criteria:

Confirmed GLA mutation predicted to be responsive to migalastat in the human embryonic kidney (HEK-293) cell-based assay

16-74 years of age

Strong clinical indication for treatment of Fabry disease

No other treatment option including either unsuitable for ERT or unable to access ERT

Appropriate female and male contraception

Willing to receive treatment with migalastat HCl via this program including having signed an authorization for sharing clinical data

The following are exclusion criteria, meaning that migalastat will not be approved in the following:

Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute/m2

Scheduled for renal or other organ transplant or replacement therapy

Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta) or REPLAGAL™ (agalsidase alpha)

Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol

Treated with another investigational drug within 30 days of start of migalastat HCl treatment

Unable to comply with requirements or deemed otherwise unsuitable for study entry in the opinion of the requesting physician.

Serious Adverse Events (SAEs) - All SAEs must be reported to PhV.Migalastat_SO@quintiles.com within 24 hours of any knowledge of the event.

Non-Serious Adverse events (AE) and Product Complaints (PC) must be reported within 24 hours of any knowledge of the event to PhV.Migalastat_SO@quintiles.com

Pregnancy exposure, lactation exposures and other Special Circumstance events must be reported within 24 hours of any knowledge of the event to PhV.Migalastat_SO@quintiles.com

Available in These Countries:

Canada

Chile

Egypt

India

Namibia

New Zealand

Turkey

Angola
ATB200 / AT2221 (cipaglucosidase alfa / miglustat)
ATB200 / AT2221 (cipaglucosidase alfa / miglustat)

ATB200 is a novel rhGAA currently being developed by Amicus Therpeutics, Inc. as a potential next-generation ERT for the treatment of adult patients with Pompe disease. Physician inquiries should be directed to Early Access Care at ATB200.NamedPatient@earlyaccesscare.com or 1-203-441-7938 to speak with one of our coordinators.

Serious Adverse Events (SAEs) must be faxed to +1 646-963-2056 within 24 hours of any site staff knowledge of the event. The form for reporting of SAEs is here.

Available in These Countries:

United States of America

Croatia

Germany

Italy

Portugal

Taiwan

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

Amicus Expanded Access Policy