Your first 90 days
Your first 90 days on Pombiliti + Opfolda for late-onset Pompe disease
What happens before the first fill, the days that matter, the label's rules and who to call, from the label, the drugmaker's own program and one insurer's written rules.
Pombiliti + Opfolda is a treatment day every 2 weeks, with capsules, a fasting window and an IV infusion in a set order. The first 90 days hold 7 treatment days, and this page lays out that stretch from both FDA labels, the Amicus Assist program and one insurer's written rules.
What has to happen before the first bottle ships
There is no REMS, so the first treatment day waits on an insurance approval, a pregnancy check where it applies, and an infusion site ready for a severe allergic reaction. These are the 8 things in the way, in order.
The approved group
Both labels cover adults with late-onset Pompe disease who weigh 40kg (about 88 lb) or more.
- They must also not be improving on their current enzyme replacement therapy.Pombiliti FDA label
A confirmed diagnosis
The Cigna policy shown below asks for a lab test showing low acid alpha-glucosidase (GAA) enzyme activity, or a genetic test showing changes in both copies of the GAA gene.Cigna Pombiliti policy
A record that the current treatment is not helping
The Cigna policy asks for no improvement in measured results after at least 1 year on Lumizyme or Nexviazyme.
- Its examples are a breathing test and a 6-minute walk test.Cigna Pombiliti policy
A pregnancy check
Both labels tell doctors to check for pregnancy before the first dose in anyone who could become pregnant, because the pair is not to be used during pregnancy.Pombiliti FDA label
Your weight and your kidney function
Your weight sets both doses, and the Opfolda label gives a lower capsule dose for moderate or severe kidney problems.
- Its Patient Information says to tell your healthcare provider about kidney problems.Opfolda FDA label
A 2 week gap after your last infusion
Both labels say to start 2 weeks after the last dose of the earlier enzyme replacement therapy, which Amicus describes as your next scheduled treatment day.Pombiliti + Opfolda how to take page
A referral to Amicus Assist
Your doctor's office faxes a Patient Referral Form to Amicus Assist.
- A Case Manager then contacts you and confirms whether your plan needs a prior authorization.Pombiliti + Opfolda support page (HCP)
A route for both medicines
Depending on where you are treated, Amicus Assist sends the form to a specialty pharmacy or reviews direct ordering with your prescriber.Pombiliti + Opfolda support page (HCP)
The days that matter
Counted from the first treatment day, on the every 2 weeks schedule that both labels set. Type your first-dose date and the tiles become real dates you can add to your calendar.
The tiles below count days from a first dose. Enter the date of yours, or the date you expect, and each tile becomes a real date on your calendar.
The date stays on your device. It is not sent to Trial Friend or anyone else. The calendar file works with Apple, Google and Outlook calendars.Referral form and the insurance check
Your doctor's office sends the Patient Referral Form to Amicus Assist, and a Case Manager checks your coverage.
- No source sets how long this takes, because each insurer works on its own timeline.
Approval, then delivery and booking
Amicus Assist says it helps manage delivery of both medicines based on where you are treated, and a Case Manager explains possible financial help.
First treatment day
Fasting starts at least 2 hours before the Opfolda capsules, and the Pombiliti infusion starts about 1 hour after them and takes about 4 hours.
Treatment day 2
The infusion starts at 1mg/kg per hour and, if there are no reactions, rises by 2mg/kg per hour every 30 minutes, up to 7mg/kg per hour.
Treatment day 3
If the infusion cannot start within 3 hours of the capsules, the label says to rebook both medicines.
- The new day is at least 24 hours after Opfolda was last taken.
Treatment day 4
Opfolda comes in bottles of 4, 24 or 100 capsules.
- A 4-capsule bottle holds one 260mg dose, so refill timing depends on the bottle size you are sent.
Treatment day 5
People with a new illness on infusion day may be at greater risk of infusion reactions, the label's boxed warning says.
- It adds that advanced Pompe disease can raise the risk of severe complications from a reaction.
Treatment day 6
For anyone who could become pregnant, the label calls for effective birth control during treatment and for at least 60 days after the last dose.
Treatment day 7
This is the seventh treatment day counted from day 1.
- In the main trial, breathing and walking were measured at week 52, well past this point.
The label, revision by revision
The FDA lists which sections changed in each revision of a label. These are the revisions the current Pombiliti + Opfolda label records, dated by the FDA, with a plain note on what each one meant for a patient. Older references and handouts can lag these by months.
Current label dated August 12, 2026, read from the FDA's label database today. Pombiliti FDA label
- August 2026
DailyMed posted version 8 of the Pombiliti label, whose text is still the July 2024 revision and lists no recent major changes.Pombiliti FDA label
- April 2026
BioMarin said it completed its purchase of Amicus Therapeutics, though both labels and the Amicus Assist pages still carry the Amicus name.BioMarin press release
- November 2024
The current Opfolda label on DailyMed, version 5, carries this date, and its text is the July 2024 revision.Opfolda FDA label
- July 2024
The FDA added sleepiness to the side effects listed for Opfolda. It also added lab findings on how miglustat interacts with the body's drug transporters.FDA letter
- September 2023
The FDA approved Opfolda capsules on the same day, and required a heart rhythm study and lab studies of drug interactions after approval.FDA letter
How the label says to take it
The parts of the prescribing information and the patient leaflet that come up in the first 3 months, in plain words.
- Two medicines, always together
Pombiliti must be given with Opfolda, and if the Opfolda dose is missed, the label says the Pombiliti infusion should not be given.Pombiliti FDA label
- Opfolda capsules by weight
People who weigh 50kg (about 110 lb) or more take 260mg, which is 4 capsules of 65mg.
- People from 40kg to under 50kg (about 88 to 110 lb) take 195mg, which is 3 capsules.Opfolda FDA label
- Kidney problems change the Opfolda dose
With moderate or severe kidney impairment, the dose is 195mg at 50kg (about 110 lb) or more, and 130mg from 40kg to under 50kg.
- Mild impairment does not change the dose, and the label says the effect of end stage kidney disease on Opfolda is unknown.Opfolda FDA label
- Fasting around the capsules
No food or other drinks for at least 2 hours before and 2 hours after Opfolda.
- Unsweetened drinks are allowed, such as water, or tea or coffee with no cream, sugar or sweeteners.Opfolda FDA label
- How Opfolda is swallowed
Capsules are swallowed whole, only with an unsweetened drink, about 1 hour before the Pombiliti infusion starts.Opfolda FDA label
- The Pombiliti dose
The dose is 20mg/kg of actual body weight, given into a vein every 2 weeks.
- Each vial holds 105mg, and the infusion bag holds 250mL to 700mL depending on weight.Pombiliti FDA label
- Infusion speed
Every infusion starts at 1mg/kg per hour, and if there are no reactions staff raise it by 2mg/kg per hour every 30 minutes, up to 7mg/kg per hour.
- The whole infusion takes about 4 hours, the label says.Pombiliti FDA label
- The whole treatment day
Amicus tells infusion staff the entire process takes about 7 hours, from the start of the fast to the end of the infusion.Guide to Getting Started (HCP)
- If the infusion is delayed
The infusion should start no later than 3 hours after the capsules.
- Past that point, both medicines are rebooked for at least 24 hours after Opfolda was last taken.Pombiliti FDA label
- If Opfolda is missed
Without the Opfolda dose, Pombiliti should not be given, and the Patient Information says to call your healthcare provider right away to reschedule the infusion.Opfolda FDA label
- If a whole treatment day is missed
If both medicines are missed, both labels say to restart treatment as soon as possible.Pombiliti FDA label
- Medicines before an infusion
The label tells doctors to consider allergy, fever or steroid medicines before each infusion.
- People who needed them with their earlier enzyme therapy get them before Pombiliti too.Pombiliti FDA label
- If a reaction happens
For a mild or moderate reaction, staff may pause or slow the infusion and treat the symptoms.
- For a severe reaction, the label says to stop Pombiliti right away and treat it.Pombiliti FDA label
- Pregnancy
Both labels say the pair must not be used during pregnancy, because animal studies point to possible harm to an unborn baby.
- The labels add that there are no data from human pregnancies.Pombiliti FDA label
- Birth control
People who can become pregnant should use effective birth control during treatment and for at least 60 days after the last dose, both labels say.Pombiliti FDA label
- Breastfeeding
Both labels say breastfeeding is not recommended during treatment, and the Patient Information suggests talking with your healthcare provider about the best way to feed your baby.Opfolda FDA label
- Fertility
Based on animal studies, the labels say the pair may affect fertility in women and men.
- The Patient Information says to raise any concerns with your healthcare provider.Opfolda FDA label
- Opfolda is approved only with Pombiliti
Opfolda capsules hold 65mg of miglustat, and the label approves them only in combination with Pombiliti, for late-onset Pompe disease.Opfolda FDA label
- Storing Opfolda
Opfolda is kept at room temperature, 68°F to 77°F (20°C to 25°C), in its original container to protect it from light.Opfolda FDA label
What the label says to act on
The symptoms the label singles out, and what it tells patients and prescribers to do about each. If any of these happen, the label's instruction is the one to follow, with your care team.
Signs of a severe allergic reaction
Trouble breathing, rash, low blood pressure, tight airways, swelling, a swollen throat or a swollen tongue.
- The label says to seek medical care if signs appear.Pombiliti FDA label
Infusion reactions
These can happen during the infusion or within a few hours after it, and are more likely at faster speeds.
- Across the studies in the label, 32% of people had an infusion reaction.Pombiliti FDA label
What severe infusion reactions looked like
In studies they included a swollen throat, hives, itching, chills, trouble breathing and flushing, and most infusion reactions were mild to moderate.Pombiliti FDA label
Heart or lung strain from the fluid
People with weak heart or lung function, or who hold on to fluid, may get worse during an infusion.
- The label calls for closer checks of vital signs in these people.Pombiliti FDA label
Being sick on infusion day
People with a new illness at the time of an infusion may be at greater risk of reactions, the label says.
- Advanced Pompe disease can weaken the heart and lungs, which may raise the risk of severe complications.Pombiliti FDA label
Other side effects
Across 3 studies, the most common were headache, diarrhea, tiredness, nausea, stomach area pain and fever.
- In the main trial, headache (8.2%) and diarrhea (5.9%) were the most common.Pombiliti FDA label
A possible pregnancy
The Opfolda Patient Information says to tell your healthcare provider right away if you become pregnant or think you might be pregnant.Opfolda FDA label
Why the pharmacy or the insurer says no, and what fixes it
Most first-fill delays are one of these. Knowing which one tells you who to call.
- Prior authorization pending or deniedThe Cigna policy below asks for a lab report showing low GAA enzyme activity in blood, skin cells or muscle, or a genetic test showing changes in both GAA genes.Cigna Pombiliti policy
- No record that the current treatment is not workingCigna asks that measured results, such as forced vital capacity or the 6-minute walk test, have not improved after at least 1 year on Lumizyme or Nexviazyme.Cigna Pombiliti policy
- Only one of the 2 medicines is approvedCigna reviews Pombiliti and Opfolda under separate policies with matching criteria. Each policy requires that the medicine be used with the other one.Cigna Opfolda policy
- Age or weight outside the policyCigna approves adults 18 or older who weigh more than 40kg (about 88 lb), while the label's own wording is 40kg or more.Cigna Pombiliti policy
- The doctor is not a specialistCigna asks that each medicine be prescribed by, or with advice from, a geneticist, neurologist, metabolic specialist or lysosomal storage disorder specialist.Cigna Pombiliti policy
- A dose outside the plan's limitCigna approves up to 20mg/kg of Pombiliti, no more often than once every 2 weeks, and other doses are reviewed case by case.Cigna Pombiliti policy
- A pharmacy that cannot fill itAmicus lists a limited specialty pharmacy network of Accredo, CVS Specialty and Orsini. It also lists specialty distributors for sites that order directly.Product ordering information
- A copay you cannot payCommercial or private insurance: Amicus says eligible patients may pay as little as $0 per month, up to an annual maximum. The offer excludes Medicare, Medicaid, Veterans Administration, TRICARE and similar federal or state programs.Pombiliti + Opfolda support page (HCP)
- Infusion costs in Rhode IslandAmicus says its co-pay help does not cover infusion-related costs for people who live in Rhode Island, in line with state law.Pombiliti + Opfolda support page (HCP)
- No insurance, or not enough coverageThe Amicus Patient Assistance Program provides free product to eligible uninsured or underinsured people, and Amicus Assist gives details at 1-833-264-2872.Pombiliti + Opfolda support page (HCP)
Cigna prior authorization policy, August 15, 2026
- Patient is 18 years of age or older
- Patient weighs more than 40kg (about 88 lb), as the policy words it
- Pombiliti will be used in combination with Opfolda
- No improvement in objective measures, such as forced vital capacity or the 6-minute walk test, after at least 1 year on Lumizyme or Nexviazyme
- Late-onset Pompe disease shown by low acid alpha-glucosidase activity in blood, skin cells or muscle, or by disease-causing variants in both GAA genes, with documents
- Prescribed by, or with advice from, a geneticist, neurologist, metabolic disorder specialist or lysosomal storage disorder specialist
- Each dose no more than 20mg/kg by IV, no more often than once every 2 weeks
Approval length. One year.
This policy (Drug Coverage Policy IP0591) covers Pombiliti and added documentation requirements on August 15, 2026. Cigna reviews Opfolda under a matching policy, IP0598, effective July 15, 2026. IP0591 cites the July 2024 Pombiliti label, which is still the current text. It is here as an example of what a plan asks for, not as a rule. Cigna Pombiliti policy
Who to call, and what they can do
The drugmaker's program handles most of the first-fill problems above. Charity funds and foundation help are on the help paying page.
- A Case Manager who confirms whether a prior authorization is needed and explains eligibility and possible financial help
- A Patient Education Liaison who can help you prepare for each treatment day Pombiliti + Opfolda patient support page
- Co-pay assistance for eligible people with commercial or private insurance, who may pay as little as $0 per month, up to an annual maximum
- Co-pay help is not valid with Medicare, Medicaid, Veterans Administration, TRICARE, Department of Defense or similar federal or state programs
- The Amicus Patient Assistance Program, free product for eligible uninsured or underinsured people
- Help finding independent charitable foundations, which set their own rules and decide their own support
- Spanish-speaking Case Managers at the same number Product ordering information
- The MyDay Pompe app for treatment day, from the App Store or Google Play Pombiliti + Opfolda patient support page
Details as the drugmaker publishes them. Pombiliti + Opfolda support page (HCP)
Five questions for the pharmacist or the support line
- Who sends my Opfolda and my Pombiliti, and where is each one delivered?
- How many Opfolda capsules make up my dose, and which bottle size will I get?
- What time do I stop eating, and what time do I take the capsules on treatment day?
- What happens if my infusion starts late after I have taken Opfolda?
- Which medicines will I get before my infusion, and why?
- Has my kidney function been checked against my Opfolda dose?
- Has co-pay help been applied to both medicines and to the infusion costs?
Questions people ask in the first weeks
How long does a Pombiliti + Opfolda treatment day take?
The infusion takes about 4 hours, and Amicus tells infusion staff the entire process takes about 7 hours, from the start of the fast to the infusion's end.
What can I eat or drink around the Opfolda capsules?
No food or other drinks for at least 2 hours before and 2 hours after the capsules. Unsweetened drinks are allowed, such as water, or tea or coffee with no cream, sugar or sweeteners.
How many Opfolda capsules will I take?
The label dose is 260mg (4 capsules) at 50kg (about 110 lb) or more, and 195mg (3 capsules) from 40kg to under 50kg. Kidney problems can lower the dose, and your prescriber sets it.
What if my infusion is delayed after I take Opfolda?
The label says the infusion should start no later than 3 hours after the capsules. Past that, both medicines are rebooked for at least 24 hours after Opfolda was last taken.
What if I miss my Opfolda dose?
The label says Pombiliti should not be given without it. The Patient Information says to call your healthcare provider right away to reschedule the infusion.
Does Pombiliti have a boxed warning?
Yes, Pombiliti has carried a boxed warning since its approval in September 2023. It covers severe allergic reactions including anaphylaxis, severe infusion reactions, and sudden heart or lung failure in people at risk from the extra fluid.
Can Pombiliti + Opfolda be used during pregnancy?
No, both labels list pregnancy as a reason the pair must not be used. They call for effective birth control during treatment and for at least 60 days after the last dose.
Is Opfolda the same as other miglustat capsules?
Opfolda holds 65mg of miglustat per capsule, and its label approves it only with Pombiliti for late-onset Pompe disease. The label does not discuss using any other miglustat product in its place.
What did the main trial show?
In a 52-week trial of 123 adults, sitting FVC, a lung function measure, changed by -1.1% with Pombiliti + Opfolda and -3.3% with the comparison treatment. The comparison was an alglucosidase alfa product not approved in the U.S. The label says no conclusion can be drawn about the U.S.-approved product from this trial.
How long does it take to start after it is prescribed?
No source gives a fixed number of days, because the referral form, the prior authorization and delivery each take their own time. Both labels set the start at 2 weeks after your last earlier infusion.
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Sources
- POMBILITI (cipaglucosidase alfa-atga) for injection, for intravenous use, prescribing information, Revised: 7/2024 (SPL version 8, effective August 12, 2026), Amicus Therapeutics US, LLC, via DailyMed and openFDA, August 12, 2026.
- OPFOLDA (miglustat) capsules, for oral use, prescribing information and Patient Information, Revised: 7/2024 (SPL version 5, effective November 27, 2024), Amicus Therapeutics US, LLC, via DailyMed and openFDA, November 27, 2024.
- POMBILITI (cipaglucosidase alfa-atga) for injection, for intravenous use, prescribing information, Initial U.S. Approval: 2023, FDA, Drugs@FDA, September 28, 2023.
- How To Take - Pombilitiopfolda Patient, Amicus Therapeutics, July 1, 2026.
- Support + Resources / Support And Tools - Pombilitiopfolda Patient, Amicus Therapeutics, July 1, 2026.
- Support + Resources - Pombilitiopfolda HCP, Amicus Therapeutics, February 1, 2026.
- Guide to Getting Started: Treatment Day Checklist and Frequently Asked Questions for Healthcare Providers (PP-AT-US-0004-0125), Amicus Therapeutics, February 1, 2025.
- POMBILITI + OPFOLDA Product Ordering Information (PP-AT-US-0017-0523), Amicus Therapeutics, January 1, 2023.
- Pompe Disease – Enzyme Replacement Therapy – Pombiliti, Drug Coverage Policy IP0591, Cigna, August 15, 2026.
- Pompe Disease – Enzyme Stabilization Therapy – Opfolda, Drug Coverage Policy IP0598, Cigna, July 15, 2026.
- BioMarin Completes Acquisition of Amicus Therapeutics, BioMarin Pharmaceutical Inc., via BioSpace press releases, April 27, 2026.
- BLA 761204/S-004 supplement approval letter, Pombiliti (cipaglucosidase alfa-atga), FDA, July 25, 2024.
- NDA 215211/S-002 supplement approval letter, Opfolda (miglustat) capsules, FDA, July 25, 2024.
- BLA 761204 approval letter, Pombiliti (cipaglucosidase alfa-atga), FDA, September 28, 2023.
- NDA 215211 approval letter, Opfolda (miglustat) capsules, FDA, September 28, 2023.
This page explains what the FDA label, the drugmaker's support program and an insurer policy say. It is not medical advice and does not tell anyone to start, stop or change a medicine. Dosing, test timing and restarts after a gap are decisions for the prescribing neurologist. Found an error? Use the report link in the footer and it will be checked against the source.
The author has done professional work for companies whose products are discussed in this article. No company reviewed, paid for or had any input into it.