Nexviazyme (avalglucosidase alfa)
An approved treatment for Pompe Disease.
The same compound appears under different names depending on the context. Here is how to identify Avalglucosidase alfa wherever you encounter it, plus the key facts at a glance.
- Generic name
- Avalglucosidase alfa
- Brand name
- Nexviazyme
- Development codes
- neoGAA, GZ402666
- Drug class
- Enzyme replacement therapy
- Manufacturer
- Sanofi Genzyme
- How it's taken
- Given as an intravenous infusion every 2 weeks.
A next-generation enzyme replacement therapy for Pompe disease designed to deliver more enzyme into muscle cells than the original treatment. Features enhanced mannose-6-phosphate receptor targeting for better cellular uptake.
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Where Avalglucosidase alfa fits
Next-generation ERT for Pompe disease with improved cellular uptake compared to alglucosidase alfa. Designed to deliver more enzyme to affected muscle cells.
How Avalglucosidase alfa works
In Pompe disease, the body lacks acid alpha-glucosidase (GAA), an enzyme that breaks down glycogen inside cells. Without it, glycogen builds up in muscles and damages them. Nexviazyme is an engineered version of GAA with extra mannose-6-phosphate sugar tags on its surface.
These tags act like keys that open the doors on muscle cells more efficiently, delivering more enzyme inside where it's needed.
Mechanism: Next-generation enzyme replacement therapy with enhanced cellular uptake for Pompe disease
Side effects and safety
Nexviazyme has a boxed warning for severe allergic reactions (including anaphylaxis), infusion reactions, and a risk of sudden heart or lung failure during the infusion in people with weak heart or lung function or who are prone to fluid overload. Infusions are given where emergency equipment is available. The most common side effects include headache, tiredness, diarrhea, nausea, joint pain, dizziness, muscle pain, itching, vomiting, shortness of breath, skin redness, tingling, and hives. Some patients develop anti-drug antibodies. Pre-medication may be recommended.
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 Avalglucosidase alfa
Given as an intravenous infusion every 2 weeks. The dose is 20 mg/kg for people who weigh 30 kg or more and 40 mg/kg for those under 30 kg. Infusions usually take about 4 to 7 hours. Administered at infusion centers, with home infusion possible after initial treatments.
Availability and cost
Only available as the brand-name product.
Next-generation enzyme replacement therapy with enhanced mannose-6-phosphate targeting for improved cellular uptake. Complex biologic requiring biweekly IV infusions for a rare lysosomal storage disorder.
Help paying for Nexviazyme
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
Copay Assistance Program covers 100% of eligible out-of-pocket NEXVIAZYME drug costs up to a program maximum. Excludes Medicare, Medicaid, VA, DoD, TRICARE.
For: private insurance · source - Infusion cost help
The copay program also covers certain infusion-related costs (mixing, administration, supplies). Not covered for residents of MA or RI.
For: private insurance · source - Bridge or quick-start supply
QuickStart provides NEXVIAZYME at no charge to eligible commercially insured patients during authorization review or coverage delays or denials, typically up to 4 infusions.
For: private insurance · source - Free medicine program
Patient Assistance Program may provide NEXVIAZYME to patients with no insurance or whose insurance denied coverage; apply with your doctor.
For: no insurance, underinsured · source - Insurance and case manager help
Patient Education Liaison and Case Manager help with benefits, insurance and financial resources.
The official page does not say who qualifies. Ask the program. · source
Good to know: Detailed program terms are on Sanofi's official NEXVIAZYME HCP page; the patient page confirms CareConnectPSS support. Call 1-800-745-4447, option 3.
- 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 You are here | 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
The COMET Phase 3 trial showed Nexviazyme was at least as good as (noninferior to) alglucosidase alfa for breathing function after 49 weeks. Breathing improved slightly more with Nexviazyme (forced vital capacity rose by 2.9 vs 0.5 percentage points of the predicted value), but the trial did not prove it was better (superiority was not reached). Long-term NEO-EXT data (8 years) demonstrates durable benefit. Approved for patients 1 year and older with late-onset Pompe disease.
Development history
Approved by FDA in August 2021 as the first new Pompe treatment in 15 years. Designed to overcome the limitations of first-generation ERT by improving enzyme delivery into cells. Represents the next evolution of Pompe disease treatment.
Explore Pompe Disease trials
Other Pompe Disease treatments
Common questions about Avalglucosidase alfa
▸What is Avalglucosidase alfa (Nexviazyme)?
A next-generation enzyme replacement therapy for Pompe disease designed to deliver more enzyme into muscle cells than the original treatment. Features enhanced mannose-6-phosphate receptor targeting for better cellular uptake.
▸How does Avalglucosidase alfa work?
In Pompe disease, the body lacks acid alpha-glucosidase (GAA), an enzyme that breaks down glycogen inside cells. Without it, glycogen builds up in muscles and damages them. Nexviazyme is an engineered version of GAA with extra mannose-6-phosphate sugar tags on its surface.
These tags act like keys that open the doors on muscle cells more efficiently, delivering more enzyme inside where it's needed.
▸What are the side effects of Avalglucosidase alfa?
Nexviazyme has a boxed warning for severe allergic reactions (including anaphylaxis), infusion reactions, and a risk of sudden heart or lung failure during the infusion in people with weak heart or lung function or who are prone to fluid overload. Infusions are given where emergency equipment is available. The most common side effects include headache, tiredness, diarrhea, nausea, joint pain, dizziness, muscle pain, itching, vomiting, shortness of breath, skin redness, tingling, and hives. Some patients develop anti-drug antibodies. Pre-medication may be recommended.
▸How is Avalglucosidase alfa taken?
Given as an intravenous infusion every 2 weeks. The dose is 20 mg/kg for people who weigh 30 kg or more and 40 mg/kg for those under 30 kg. Infusions usually take about 4 to 7 hours. Administered at infusion centers, with home infusion possible after initial treatments.
▸Is Avalglucosidase alfa FDA approved?
Yes, Avalglucosidase alfa (Nexviazyme) is FDA approved (2021) for the treatment of Pompe Disease.
▸How does Nexviazyme improve on the original Pompe therapy?
Nexviazyme is engineered with extra mannose-6-phosphate sugar tags that act like keys to open muscle cell doors more efficiently, delivering more enzyme where it is needed. In the COMET trial, it was at least as good as alglucosidase alfa for breathing function, with a slightly larger improvement, but the trial did not prove it was better.
▸Who is eligible for Nexviazyme?
Nexviazyme is approved for patients 1 year and older with late-onset Pompe disease. It represents the first new Pompe treatment approved in 15 years when it received FDA approval in August 2021.
▸How long has Nexviazyme shown benefit?
Long-term NEO-EXT data spanning 8 years demonstrates durable benefit from avalglucosidase alfa treatment, including sustained improvements in respiratory function and walking ability.
▸How is Nexviazyme administered?
Nexviazyme is given as an intravenous infusion every 2 weeks, at 20 mg/kg for people who weigh 30 kg or more and 40 mg/kg for those under 30 kg. Infusions typically take several hours and are initially administered at infusion centers, with home infusion possible after initial treatments.
▸What side effects are most common with Nexviazyme?
Infusion-associated reactions are the most common, including headache, fatigue, nausea, chills, vomiting, and muscle pain. Hypersensitivity and anaphylaxis are possible. Some patients develop anti-drug antibodies. Pre-medication may be recommended.
▸How does Nexviazyme compare to Pombiliti + Opfolda?
Both are newer alternatives to the original alglucosidase alfa. Nexviazyme uses enhanced cellular targeting, while Pombiliti + Opfolda combines an improved enzyme with an oral chaperone stabilizer. Neither was proven better than the original therapy on its main trial goal, and they have not been compared with each other.