Official Title
Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease
Brief Summary

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

Detailed Description

This program is being offered on a patient by patient basis and will require company, IRB/IEC
and single patient IND approval.

Individual Patients
Pompe Disease

Biological: ATB200

Drug: AT2221

Eligibility Criteria

Inclusion Criteria:

1. Subject must have a diagnosis of Pompe disease based on documentation of one of the

1. deficiency of GAA enzyme

2. GAA genotyping

2. Patient does not currently qualify for an Amicus sponsored on-going clinical trial or
is declining on currently approved ERT (e.g. Myozyme)

3. The patient must be willing to receive treatment with ATB200/AT2221 via this program,
which includes signing an authorization form for sharing clinical data with Amicus
Therapeutics, and its agent Early Access Care LLC.

Exclusion Criteria:

1. Patient, whether male or female, is planning to conceive a child during the treatment

2. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa,
or AT2221

3. Patient has a medical or any other extenuating condition or circumstance that may, in
the opinion of the investigator, pose an undue safety risk to the subject or
compromise his/her ability to comply with or adversely impact protocol requirements.

4. Patient has received any gene therapy at any time.

5. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca),
acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)

Eligibility Gender
Eligibility Age
Minimum: N/A ~ Maximum: N/A

For Site

For Patient

Amicus Therapeutics
NCT Number
Expanded Access
Compassionate Use
MeSH Terms
Glycogen Storage Disease Type II