Starting Pombiliti + Opfolda (cipaglucosidase alfa-atga + miglustat)Your first 90 days with pompe disease: the steps, the dates and who to call

How you take it

Once every 2 weeks, you swallow Opfolda capsules, then about 1 hour later you get a Pombiliti infusion into a vein that takes about 4 hours.

Before your first dose

• A lab test or a genetic test that confirms late-onset Pompe disease, since your insurance may ask for the report.
• Your insurance may ask for test results showing your current treatment is not helping, such as a breathing test or a walk test.
• A pregnancy check before the first dose, if you could become pregnant, because these medicines must not be used during pregnancy.
• Your current weight sets both doses, and the Opfolda Patient Information says to tell your healthcare provider about kidney problems, which can lower the dose.
• Your insurance needs to approve both medicines, and Amicus Assist, the drugmaker's support program, can help at 1-833-264-2872.
• The first treatment day is booked 2 weeks after your last infusion of your earlier medicine, at a place ready to treat a serious allergic reaction.
• On treatment day, no food for 2 hours before and 2 hours after the capsules. Water, and tea or coffee with nothing added, are allowed.

The first 90 days

1. Before the first treatment dayDoctor's office sends the referral form, insurance check
2. Once approvedApproval, then delivery and booking
3. Day 1Day 1: fast, capsules, then the first infusion
4. Day 15Treatment day 2
5. Day 29Treatment day 3
6. Day 43Fourth treatment day, and a check on your Opfolda supply
7. Day 57Treatment day 5
8. Day 71Treatment day 6
9. Day 85Treatment day 7