Drug-drug Interaction Study

This was a multi-center, international, open-label, two-period, fixed-sequence crossover
study to evaluate the safety and pharmacokinetic effect of single ascending doses of
duvoglustat on rhGAA administered 1 hour before initiation of a single rhGAA infusion. During
Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each
participant received a single oral dose of duvoglustat 1 hour prior to initiation of a single
rhGAA infusion.

Inclusion Criteria:

- Male or female, diagnosed with Pompe disease and between 18 and 65 years of age,
inclusive

- Participant has been on a stable regimen and dose of rhGAA for at least 3 months
before screening (stable regimen defined as currently receiving rhGAA every 2 weeks
and stable dose defined as not varying by more than ± 10%)

- Participant has an estimated glomerular filtration rate (eGFR) ≥ 50 mL/min at
Screening; eGFR to be estimated using the 4-parameter Modification of Diet in Renal
Disease (MDRD) equation:

eGFR (mL/min/1.73 m^2) = 175 x (Scr)^(-1.154) x (Age)^(-0.203) x (0.742 if female) x (1.212
if African-American)

- Male and female participants of childbearing potential agree to use medically accepted
methods of contraception during the study and for 30 days after study completion

- Participant is willing and able to provide written informed consent and is able to
comply with all study procedures

Exclusion Criteria:

- Participant has had a documented transient ischemic attack, ischemic stroke, unstable
angina, or myocardial infarction within the 3 months before Screening

- Participant has clinically significant unstable cardiac disease (for example, cardiac
disease requiring active management, such as symptomatic arrhythmia, unstable angina,
or New York Heart Association class III or IV congestive heart failure)

- Participant requiring mechanical ventilation or is confined to a wheelchair

- Participant has a history of allergy or sensitivity to study drug (including
excipients) or other iminosugars (for example, miglustat, miglitol)

- Participant is pregnant or breastfeeding

- Participant tests positive for hepatitis B surface antigen or hepatitis C antibody

- Participant has received any investigational/experimental drug or device within 30
days of Screening

- Participant has any intercurrent illness or condition that may preclude the
participant from fulfilling the protocol requirements or suggests to the investigator
that the potential participant may have an unacceptable risk by participating in this
study