Pombiliti + Opfolda (cipaglucosidase alfa + miglustat)
An approved treatment for Pompe Disease.
The same compound appears under different names depending on the context. Here is how to identify Cipaglucosidase alfa + miglustat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Cipaglucosidase alfa + miglustat
- Brand name
- Pombiliti + Opfolda
- Development code
- ATB200/AT2221
- Drug class
- Enzyme replacement therapy + chaperone
- Manufacturer
- Amicus Therapeutics (part of BioMarin since April 2026)
- How it's taken
- Two-step process: miglustat capsule taken orally 1 hour before infusion, then cipaglucosidase alfa given as IV infusion every 2 weeks at 20 mg/kg.
A two-component therapy combining a next-generation enzyme (cipaglucosidase alfa, IV) with an oral enzyme stabilizer (miglustat) that protects the enzyme in the bloodstream, delivering more active enzyme to muscle cells.
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Where Cipaglucosidase alfa + miglustat fits
Combination ERT plus chaperone therapy for late-onset Pompe disease. The miglustat component stabilizes the enzyme during infusion, potentially improving its effectiveness in reaching target tissues.
How Cipaglucosidase alfa + miglustat works
This two-part system works like a bodyguard escort. Cipaglucosidase alfa is an improved version of the GAA enzyme with better targeting to muscle cells.
Miglustat (taken orally before the infusion) acts as a molecular chaperone that binds to the enzyme in the bloodstream and prevents it from being degraded before it reaches the muscles. More intact enzyme gets into cells, where it can break down the accumulated glycogen.
Mechanism: Two-component ERT: enhanced enzyme replacement plus oral enzyme stabilizer
Side effects and safety
Pombiliti has a boxed warning for severe allergic reactions including anaphylaxis, severe infusion-associated reactions, and a risk of sudden heart or lung failure during the infusion in people prone to fluid overload or with weak heart or lung function. The combination can harm an unborn baby and must not be used during pregnancy; use effective birth control during treatment and for at least 60 days after the last dose. Common side effects include headache, diarrhea, tiredness, nausea, belly pain, and fever. Anti-drug antibodies may develop, and high antibody levels have been linked to serious allergic and infusion reactions. Pre-medication may help with infusion reactions.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Cipaglucosidase alfa + miglustat
Two-step process: miglustat capsule taken orally 1 hour before infusion, then cipaglucosidase alfa given as IV infusion every 2 weeks at 20 mg/kg. The oral component must be taken in a fasted state. Approved for adults with late-onset Pompe weighing 40 kg or more who are not improving on their current enzyme replacement therapy.
Availability and cost
Only available as the brand-name product.
Two-component ERT combining a novel recombinant enzyme with an oral enzyme stabilizer (miglustat) to enhance enzyme activity and uptake. Dual-drug complexity adds to costs.
Help paying for Pombiliti + Opfolda
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Eligible commercially insured patients may pay as little as $0 per month. Annual maximum applies; not for Medicare, Medicaid, TRICARE or VA.
For: private insurance · source - Free medicine program
The Amicus Patient Assistance Program provides free product to eligible uninsured or underinsured patients.
For: no insurance, underinsured · source - Insurance and case manager help
Case Managers verify and explain insurance coverage, help with prior authorization and coordinate deliveries.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: Your doctor enrolls you with the Patient Referral Form. Co-pay help does not cover infusion-related costs for Rhode Island residents. Spanish-speaking case managers available.
- From a charity · TotalAssist (formerly PAN Foundation)Pompe Disease fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).
- From a charity · Muscular Dystrophy AssociationMDA Durable Medical Equipment (DME) Grant Program fundApply directly
Pays for: Medical equipment (wheelchairs, lifts, canes and other DME), up to $1,000 per year.
The foundation says: “Status not shown on page” - From a charity · The Assistance FundPompe Disease fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
How Pompe disease treatments compare
Three FDA-approved enzyme replacement therapies are available for late-onset Pompe disease. Your metabolic disease specialist will recommend a treatment based on your disease severity, current respiratory and motor function, prior ERT history, and individual response. This chart summarizes the key practical differences between options.
3 FDA-approved pompe disease treatments are available: Lumizyme (alglucosidase alfa, approved 2010); Nexviazyme (avalglucosidase alfa-ngpt, approved 2021); Pombiliti + Opfolda (cipaglucosidase alfa + miglustat, approved 2023). Lumizyme is typically used as first-generation ert; approved for iopd and lopd.
| Drug | How it works | How it’s given | How often | Where you get it | Typical use | FDA approved |
|---|---|---|---|---|---|---|
Lumizyme alglucosidase alfa | Enzyme replacement — provides the GAA enzyme your body can't make enough of to break down glycogen in muscle tissue | Intravenous infusion (20 mg/kg) | Every 2 weeks | Infusion center or home infusion | First-generation ERT; approved for IOPD and LOPD | 2010 |
Nexviazyme avalglucosidase alfa-ngpt | Enhanced enzyme replacement — same GAA enzyme with bis-mannose-6-phosphate targeting for improved cellular uptake into muscle | Intravenous infusion (20 mg/kg for patients ≥30 kg; 40 mg/kg for patients <30 kg) | Every 2 weeks | Infusion center or home infusion | Next-generation ERT; first-line or switch from Lumizyme | 2021 |
Pombiliti + Opfolda cipaglucosidase alfa + miglustat You are here | Enzyme + chaperone — ERT paired with an oral stabilizer (miglustat) that protects the enzyme from degradation in the bloodstream | Intravenous infusion (20 mg/kg) + oral capsule (65 mg taken 1 hour before infusion) | Every 2 weeks | Infusion center or home infusion | For adults ≥40 kg with LOPD not improving on current ERT (switch therapy) | 2023 |
This chart summarizes approved Pompe disease treatments to help you understand the landscape. It is not medical advice. Pombiliti + Opfolda is specifically approved for adults with late-onset Pompe disease who have previously received enzyme replacement therapy. Treatment decisions depend on your individual disease severity, respiratory function, prior ERT response, and overall health. Always discuss options with your metabolic disease specialist.
Clinical trial results
In the PROPEL Phase 3 trial against alglucosidase alfa, people already on enzyme replacement therapy who switched to this combination had numerically better breathing (FVC) and 6-minute walk results after 52 weeks; the FDA label describes these as favorable trends in that group. Four-year open-label extension (PROPEL OLE) data presented September 2025 confirmed durability.
Development history
Approved by FDA in September 2023 as the first two-component therapy for Pompe disease. Developed by Amicus Therapeutics using their proprietary chaperone-enzyme platform. Japan approval followed in June 2025. BioMarin acquired Amicus in April 2026.
Explore Pompe Disease trials
Other Pompe Disease treatments
Common questions about Cipaglucosidase alfa + miglustat
▸What is Cipaglucosidase alfa + miglustat (Pombiliti + Opfolda)?
A two-component therapy combining a next-generation enzyme (cipaglucosidase alfa, IV) with an oral enzyme stabilizer (miglustat) that protects the enzyme in the bloodstream, delivering more active enzyme to muscle cells.
▸How does Cipaglucosidase alfa + miglustat work?
This two-part system works like a bodyguard escort. Cipaglucosidase alfa is an improved version of the GAA enzyme with better targeting to muscle cells.
Miglustat (taken orally before the infusion) acts as a molecular chaperone that binds to the enzyme in the bloodstream and prevents it from being degraded before it reaches the muscles. More intact enzyme gets into cells, where it can break down the accumulated glycogen.
▸What are the side effects of Cipaglucosidase alfa + miglustat?
Pombiliti has a boxed warning for severe allergic reactions including anaphylaxis, severe infusion-associated reactions, and a risk of sudden heart or lung failure during the infusion in people prone to fluid overload or with weak heart or lung function. The combination can harm an unborn baby and must not be used during pregnancy; use effective birth control during treatment and for at least 60 days after the last dose. Common side effects include headache, diarrhea, tiredness, nausea, belly pain, and fever. Anti-drug antibodies may develop, and high antibody levels have been linked to serious allergic and infusion reactions. Pre-medication may help with infusion reactions.
▸How is Cipaglucosidase alfa + miglustat taken?
Two-step process: miglustat capsule taken orally 1 hour before infusion, then cipaglucosidase alfa given as IV infusion every 2 weeks at 20 mg/kg. The oral component must be taken in a fasted state. Approved for adults with late-onset Pompe weighing 40 kg or more who are not improving on their current enzyme replacement therapy.
▸Is Cipaglucosidase alfa + miglustat FDA approved?
Yes, Cipaglucosidase alfa + miglustat (Pombiliti + Opfolda) is FDA approved (2023) for the treatment of Pompe Disease.
▸Why does this therapy have two components?
Cipaglucosidase alfa is an improved GAA enzyme, and miglustat acts as a molecular chaperone that protects the enzyme in the bloodstream from being degraded before reaching muscle cells. Taking miglustat before the infusion ensures more active enzyme gets to where it is needed.
▸How is Pombiliti + Opfolda administered?
Miglustat (Opfolda) capsule is taken orally in a fasted state 1 hour before the infusion. Then cipaglucosidase alfa (Pombiliti) is given as an IV infusion every 2 weeks at 20 mg/kg. The two-step process is essential for optimal enzyme delivery.
▸What did the PROPEL trial show?
In the PROPEL Phase 3 trial against alglucosidase alfa, people already on enzyme replacement therapy who switched had numerically better breathing and walking results at 52 weeks. Four-year open-label extension data confirmed durability of these improvements.
▸Who is eligible for Pombiliti + Opfolda?
This therapy is approved for adults weighing 40 kg or more with late-onset Pompe disease. It provides an alternative for patients who may not be responding optimally to first-generation enzyme replacement therapy.
▸How does this therapy compare to Nexviazyme?
Both are newer alternatives to alglucosidase alfa. Pombiliti + Opfolda uses a chaperone to protect the enzyme during infusion, while Nexviazyme uses enhanced cellular targeting with extra mannose-6-phosphate tags. Head-to-head comparison data is not available.
▸Who developed Pombiliti + Opfolda?
Developed by Amicus Therapeutics using their proprietary chaperone-enzyme platform. FDA approved in September 2023 as the first two-component therapy for Pompe disease. Japan approval followed in June 2025.