Lumizyme (alglucosidase alfa)
Also marketed as Myozyme. An approved treatment for Pompe Disease.
The same compound appears under different names depending on the context. Here is how to identify Alglucosidase alfa wherever you encounter it, plus the key facts at a glance.
- Generic name
- Alglucosidase alfa
- Brand names
- Lumizyme, Myozyme
- Drug class
- Enzyme replacement therapy
- Manufacturer
- Sanofi Genzyme
- How it's taken
- Given as an intravenous infusion every 2 weeks at 20 mg/kg.
The first and foundational treatment for Pompe disease. Provides the acid alpha-glucosidase enzyme that patients' bodies cannot produce, clearing the glycogen buildup that damages muscles, the heart, and respiratory function.
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Where Alglucosidase alfa fits
First approved therapy for Pompe disease, providing the missing acid alpha-glucosidase enzyme. Transformed Pompe disease from a fatal infantile condition to a treatable chronic disease.
How Alglucosidase alfa works
Alglucosidase alfa is a manufactured version of the GAA enzyme. When infused into the bloodstream, it is taken up by cells via mannose-6-phosphate receptors and directed to lysosomes, where it breaks down the accumulated glycogen. In infantile-onset Pompe disease, it can be lifesaving by preventing fatal cardiac enlargement. In late-onset disease, it stabilizes respiratory and motor function.
Mechanism: Enzyme replacement therapy providing acid alpha-glucosidase for Pompe disease
Side effects and safety
Lumizyme has a boxed warning for 3 risks: life-threatening allergic reactions including anaphylaxis; immune reactions that can affect the kidneys and skin; and sudden heart or breathing failure in infants whose heart or lungs are already weak, from the fluid given with the infusion. Common infusion reactions include fever, rash, flushing, hives, headache, and changes in heart rate or oxygen levels. Antibody development may reduce efficacy over time. In infants with Pompe disease and an enlarged heart, dangerous heart rhythm problems, including cardiac arrest and death, have happened during general anesthesia to place a central IV line for infusions, so extra care is needed if anesthesia is planned.
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 Alglucosidase alfa
Given as an intravenous infusion every 2 weeks at 20 mg/kg. Infusions can take 4+ hours. In the US it is sold as Lumizyme, which since August 2014 has been approved for all patients with Pompe disease. Myozyme, the original US brand approved in 2006, is no longer made in the US and is the brand name used in other countries.
Availability and cost
Only available as the brand-name product.
First enzyme replacement therapy for Pompe disease, a recombinant human acid alpha-glucosidase requiring biweekly IV infusions. Orphan biologic for a rare lysosomal storage disorder.
Help paying for Lumizyme
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.
- Insurance and case manager help
Sanofi's CareConnectPSS offers a dedicated team for people with Pompe disease: disease education, insurance help and financial assistance options.
The official page does not say who qualifies. Ask the program. · source - Copay help
CareConnect Copay Program may help with drug costs and certain infusion charges up to a program maximum for certain Sanofi therapies. Excludes Medicare, Medicaid, TRICARE.
For: private insurance · source - Free medicine program
CareConnect Patient Assistance Program may give certain Sanofi therapies free to eligible patients without insurance or whose plan won't cover them, temporarily.
For: no insurance, underinsured · source
Good to know: Lumizyme's patient site points to CareConnectPSS. Sanofi's Lumizyme HCP page lists the CareConnect Copay Program application. No official patient page states Lumizyme-specific copay or free-drug terms, so confirm eligibility by calling 1-800-745-4447, option 3. Myozyme is the non-US brand name.
- 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 You are here | 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 | 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
Pivotal trials in infantile-onset Pompe showed dramatic improvement in survival and cardiac function. Late-onset studies showed stabilization of walking ability and lung function. Over 15 years of real-world data confirm long-term benefit.
Development history
Myozyme approved by FDA in April 2006 for infantile-onset Pompe disease. Lumizyme approved in May 2010 for late-onset, then extended in August 2014 to all patients with Pompe disease; Myozyme is no longer made in the US. One of the landmark rare disease enzyme replacement therapies, transforming Pompe disease from a uniformly fatal infantile condition to a manageable chronic disease.
Explore Pompe Disease trials
Other Pompe Disease treatments
Common questions about Alglucosidase alfa
▸What is Alglucosidase alfa (Lumizyme)?
The first and foundational treatment for Pompe disease. Provides the acid alpha-glucosidase enzyme that patients' bodies cannot produce, clearing the glycogen buildup that damages muscles, the heart, and respiratory function.
▸How does Alglucosidase alfa work?
Alglucosidase alfa is a manufactured version of the GAA enzyme. When infused into the bloodstream, it is taken up by cells via mannose-6-phosphate receptors and directed to lysosomes, where it breaks down the accumulated glycogen. In infantile-onset Pompe disease, it can be lifesaving by preventing fatal cardiac enlargement. In late-onset disease, it stabilizes respiratory and motor function.
▸What are the side effects of Alglucosidase alfa?
Lumizyme has a boxed warning for 3 risks: life-threatening allergic reactions including anaphylaxis; immune reactions that can affect the kidneys and skin; and sudden heart or breathing failure in infants whose heart or lungs are already weak, from the fluid given with the infusion. Common infusion reactions include fever, rash, flushing, hives, headache, and changes in heart rate or oxygen levels. Antibody development may reduce efficacy over time. In infants with Pompe disease and an enlarged heart, dangerous heart rhythm problems, including cardiac arrest and death, have happened during general anesthesia to place a central IV line for infusions, so extra care is needed if anesthesia is planned.
▸How is Alglucosidase alfa taken?
Given as an intravenous infusion every 2 weeks at 20 mg/kg. Infusions can take 4+ hours. In the US it is sold as Lumizyme, which since August 2014 has been approved for all patients with Pompe disease. Myozyme, the original US brand approved in 2006, is no longer made in the US and is the brand name used in other countries.
▸Is Alglucosidase alfa FDA approved?
Yes, Alglucosidase alfa (Lumizyme) is FDA approved (2006) for the treatment of Pompe Disease.
▸How did alglucosidase alfa transform Pompe disease?
Before alglucosidase alfa, infantile-onset Pompe disease was uniformly fatal, typically within the first year of life. Its approval in 2006 transformed Pompe from a fatal condition to a manageable chronic disease, dramatically improving survival in infants and stabilizing function in adults.
▸What is the difference between Myozyme and Lumizyme?
Both are alglucosidase alfa from the same company, made at different manufacturing scales. Myozyme was approved in the US in 2006 and Lumizyme in 2010 for late-onset Pompe disease. In August 2014 the FDA extended Lumizyme to all patients with Pompe disease, and Myozyme is no longer made in the US. Myozyme is still the brand name used outside the US.
▸What are CRIM-negative patients?
CRIM (Cross-Reactive Immunologic Material) negative patients produce no endogenous GAA enzyme at all, making them more likely to develop severe immune responses to alglucosidase alfa. These patients may need immune tolerance protocols before starting ERT.
▸How long do infusions take?
Infusions of alglucosidase alfa can take 4 or more hours and are given every 2 weeks at 20 mg/kg body weight. The long infusion time is one reason newer therapies with improved delivery have been developed.
▸Should patients switch to newer Pompe therapies?
Some patients stable on alglucosidase alfa may benefit from switching to Nexviazyme or Pombiliti + Opfolda, which showed advantages in clinical trials. However, switching decisions should be individualized based on response, tolerance, and disease status.
▸What long-term outcomes have been seen?
Over 15 years of real-world data confirm long-term benefit including sustained improvements in survival and cardiac function in infantile patients, and stabilization of walking ability and lung function in late-onset patients.