Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Inclusion criteria

• Patient has been diagnosed with Pompe Disease prior to or during the screening period based on 2 GAA gene mutations and either: endogenous GAA activity 10% reduction in supine FVC compared to upright FVC during the Screening Period;
• Patient is naïve to Enzyme Replacement Therapy (ERT) with rhGAA;
• Patient must be able to ambulate at least 40 meters (131.2 feet) on the 6MWT conducted at the Screening visit (use of assistive devices such as walker, cane, or crutches, is permitted); and
• If subject was female, she was not lactating

Exclusion criteria

• Patient has a history of diabetes or other disease known to cause hypoglycemia and is currently receiving, or might anticipate receiving, hypoglycemic agents during the course of the study;
• Patient has been on any immunosuppressive medication other than glucocorticosteroids within 1 year prior to enrollment into this study;
• Patient requires invasive ventilatory assistance at the time of enrollment into the study;
• Patient has received any investigational medication within 30 days prior to the first dose of study drug or is scheduled to receive any investigational drug other than BMN 701 during the course of the study;
• Patient has previously been admitted to the study;
• Patient is breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
• Patient has a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient's ability to comply with the protocol requirements or compromise the patient's well being or safety;
• Patient has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.