Neurology
Pompe disease: the approved drugs compared for prescribers
Lumizyme, Nexviazyme, Pombiliti + Opfolda side by side on what each first fill waits on, what the label requires you to monitor and what an insurer asks to see, with FDA decisions, label changes and open trials for Pompe disease.
When to suspect Pompe disease and what to test →The 3 Pompe disease prescribing guides
One page per drug with the dispensing gate, prior authorization documentation, dosing, monitoring and interactions, each line sourced.
The drugs side by side
Each cell is a summary of that drug's prescribing guide, which carries the full detail and a source on every line. Insurer thresholds are one public policy shown as an example; plans differ.
| Lumizymealglucosidase alfa | Nexviazymeavalglucosidase alfa-ngpt | Pombiliti + Opfoldacipaglucosidase alfa-atga + miglustat | |
|---|---|---|---|
| Label dated | January 10, 2025 | June 5, 2025 | August 12, 2026 |
| Starting dose | 20mg/kg body weight every 2 weeks by IV infusion, IOPD and LOPD. Total volume over approximately 4 hours. | 30kg or more: 20mg/kg of actual body weight every 2 weeks IV. Under 30kg: 40mg/kg every 2 weeks. | 20mg/kg of actual body weight IV every other week over approximately 4 hours. Must be given with Opfolda. |
| Dispensing gate | No REMS (released August 1, 2014). Boxed warning: initiate in a healthcare setting with monitoring and access to CPR equipment. Prior authorization and a site of care before the first infusion; CRIM assessment early in IOPD | No REMS. Boxed warning: CPR equipment and monitoring readily available at every infusion. Prior authorization, or QuickStart for eligible commercial patients, and a site of care before the first infusion | No REMS. Boxed warning: CPR equipment and medical support readily available at every infusion. Verify pregnancy status before initiating (contraindicated in pregnancy). Prior authorization for both products and a site of care before the first treatment day |
| Required monitoring | Supervised infusion with anaphylaxis readiness; vital signs at the end of each rate step; baseline ADA, then every 3 months (example) in year 1 for IOPD or within six months for LOPD; periodic urinalysis; extra monitoring in IOPD with cardiac or respiratory compromise | Supervised infusion with anaphylaxis readiness at every dose; consider premedication; more frequent vitals in patients prone to fluid overload or with compromised cardiorespiratory function; higher ADA titers linked to more IARs | Supervised infusion with anaphylaxis readiness at every dose; consider premedication, and pretreat if it was used with prior ERT; more frequent vitals in patients prone to fluid overload or with compromised cardiorespiratory function; CLcr for the Opfolda dose |
| Example PA threshold | Cigna example: Pompe disease; deficient GAA activity in blood, fibroblasts or muscle, or biallelic pathogenic or likely pathogenic GAA variants [documentation required]; specialist prescriber; dose not above 20mg/kg IV, no more often than every 2 weeks | Cigna example: age 1 year or older; documented LOPD; deficient GAA activity in blood, fibroblasts or muscle, or biallelic pathogenic or likely pathogenic GAA variants; specialist prescriber; 20mg/kg (30kg or more) or 40mg/kg (under 30kg) every 2 weeks | Cigna example: 18 or older; more than 40kg; used with Opfolda; no improvement in objective measures (FVC, 6MWT) after at least 1 year on Lumizyme or Nexviazyme; deficient GAA activity or biallelic pathogenic or likely pathogenic GAA variants; specialist prescriber |
| Support line | CareConnect Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM EST); Co-Pay Program 1-855-448-6520; Patient Assistance Program for uninsured or denied patients; Product Services option 1 | CareConnectPSS Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM EST); QuickStart for eligible commercial patients; Co-Pay Program 1-855-448-6520; Patient Assistance Program for uninsured or denied patients | Amicus Assist 1-833-AMICUS-A (1-833-264-2872), Monday to Friday 8AM to 8PM ET; Patient Referral Form by fax 1-833-264-2873; co-pay assistance for eligible commercial patients; Amicus Patient Assistance Program |
- Label dated
- January 10, 2025
- Starting dose
- 20mg/kg body weight every 2 weeks by IV infusion, IOPD and LOPD. Total volume over approximately 4 hours.
- Dispensing gate
- No REMS (released August 1, 2014). Boxed warning: initiate in a healthcare setting with monitoring and access to CPR equipment. Prior authorization and a site of care before the first infusion; CRIM assessment early in IOPD
- Required monitoring
- Supervised infusion with anaphylaxis readiness; vital signs at the end of each rate step; baseline ADA, then every 3 months (example) in year 1 for IOPD or within six months for LOPD; periodic urinalysis; extra monitoring in IOPD with cardiac or respiratory compromise
- Example PA threshold
- Cigna example: Pompe disease; deficient GAA activity in blood, fibroblasts or muscle, or biallelic pathogenic or likely pathogenic GAA variants [documentation required]; specialist prescriber; dose not above 20mg/kg IV, no more often than every 2 weeks
- Support line
- CareConnect Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM EST); Co-Pay Program 1-855-448-6520; Patient Assistance Program for uninsured or denied patients; Product Services option 1
- Label dated
- June 5, 2025
- Starting dose
- 30kg or more: 20mg/kg of actual body weight every 2 weeks IV. Under 30kg: 40mg/kg every 2 weeks.
- Dispensing gate
- No REMS. Boxed warning: CPR equipment and monitoring readily available at every infusion. Prior authorization, or QuickStart for eligible commercial patients, and a site of care before the first infusion
- Required monitoring
- Supervised infusion with anaphylaxis readiness at every dose; consider premedication; more frequent vitals in patients prone to fluid overload or with compromised cardiorespiratory function; higher ADA titers linked to more IARs
- Example PA threshold
- Cigna example: age 1 year or older; documented LOPD; deficient GAA activity in blood, fibroblasts or muscle, or biallelic pathogenic or likely pathogenic GAA variants; specialist prescriber; 20mg/kg (30kg or more) or 40mg/kg (under 30kg) every 2 weeks
- Support line
- CareConnectPSS Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM EST); QuickStart for eligible commercial patients; Co-Pay Program 1-855-448-6520; Patient Assistance Program for uninsured or denied patients
- Label dated
- August 12, 2026
- Starting dose
- 20mg/kg of actual body weight IV every other week over approximately 4 hours. Must be given with Opfolda.
- Dispensing gate
- No REMS. Boxed warning: CPR equipment and medical support readily available at every infusion. Verify pregnancy status before initiating (contraindicated in pregnancy). Prior authorization for both products and a site of care before the first treatment day
- Required monitoring
- Supervised infusion with anaphylaxis readiness at every dose; consider premedication, and pretreat if it was used with prior ERT; more frequent vitals in patients prone to fluid overload or with compromised cardiorespiratory function; CLcr for the Opfolda dose
- Example PA threshold
- Cigna example: 18 or older; more than 40kg; used with Opfolda; no improvement in objective measures (FVC, 6MWT) after at least 1 year on Lumizyme or Nexviazyme; deficient GAA activity or biallelic pathogenic or likely pathogenic GAA variants; specialist prescriber
- Support line
- Amicus Assist 1-833-AMICUS-A (1-833-264-2872), Monday to Friday 8AM to 8PM ET; Patient Referral Form by fax 1-833-264-2873; co-pay assistance for eligible commercial patients; Amicus Patient Assistance Program
FDA decisions for Pompe disease
From the Trial Friend FDA decision calendar, which records each date from the sponsor's or the FDA's own announcement.
No pending or recent decisions on the calendar.
Recent label changes
What changed in each label revision, from the FDA's approval letters and the labels' own revision lists.
- August 2026Pombiliti + Opfolda. 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 2026Pombiliti + Opfolda. 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
- June 2025Nexviazyme. DailyMed posted version 4 of the label. Its recent changes list still dates the last major changes to September 2023. FDA label
- January 2025Lumizyme. DailyMed posted version 21 of the label, and its change list dates updates to the boxed warning, dosing and warnings to March and December 2024. FDA label
- December 2024Lumizyme. The FDA approved an update to how the label presents antibody information, in line with FDA guidance on that topic. FDA letter
- November 2024Pombiliti + Opfolda. The current Opfolda label on DailyMed, version 5, carries this date, and its text is the July 2024 revision. Opfolda FDA label
- July 2024Pombiliti + Opfolda. 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
- July 2024Pombiliti + Opfolda. The FDA added low blood pressure and sleepiness to the side effects listed for Pombiliti, along with wording edits. FDA letter
Open trials
Questions prescribers ask about Pompe disease
Which Pompe disease drugs require a REMS?
None of the 3 Pompe disease drugs on this page has a REMS: Lumizyme, Nexviazyme and Pombiliti + Opfolda. Each prescribing guide lists what does gate the first fill.
What monitoring do Pompe disease drugs require?
Lumizyme: Supervised infusion with anaphylaxis readiness; vital signs at the end of each rate step; baseline ADA, then every 3 months (example) in year 1 for IOPD or within six months for LOPD; periodic urinalysis; extra monitoring in IOPD with cardiac or respiratory compromise. Nexviazyme: Supervised infusion with anaphylaxis readiness at every dose; consider premedication; more frequent vitals in patients prone to fluid overload or with compromised cardiorespiratory function; higher ADA titers linked to more IARs. Pombiliti + Opfolda: Supervised infusion with anaphylaxis readiness at every dose; consider premedication, and pretreat if it was used with prior ERT; more frequent vitals in patients prone to fluid overload or with compromised cardiorespiratory function; CLcr for the Opfolda dose.
What do insurers require before covering Lumizyme, Nexviazyme and Pombiliti + Opfolda?
Each answer below is one public insurer policy, an example rather than every plan. Lumizyme: Cigna example: Pompe disease; deficient GAA activity in blood, fibroblasts or muscle, or biallelic pathogenic or likely pathogenic GAA variants [documentation required]; specialist prescriber; dose not above 20mg/kg IV, no more often than every 2 weeks. Nexviazyme: Cigna example: age 1 year or older; documented LOPD; deficient GAA activity in blood, fibroblasts or muscle, or biallelic pathogenic or likely pathogenic GAA variants; specialist prescriber; 20mg/kg (30kg or more) or 40mg/kg (under 30kg) every 2 weeks. Pombiliti + Opfolda: Cigna example: 18 or older; more than 40kg; used with Opfolda; no improvement in objective measures (FVC, 6MWT) after at least 1 year on Lumizyme or Nexviazyme; deficient GAA activity or biallelic pathogenic or likely pathogenic GAA variants; specialist prescriber.
Which support programs start Pompe disease patients on therapy?
Lumizyme: CareConnect Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM EST); Co-Pay Program 1-855-448-6520; Patient Assistance Program for uninsured or denied patients; Product Services option 1. Nexviazyme: CareConnectPSS Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM EST); QuickStart for eligible commercial patients; Co-Pay Program 1-855-448-6520; Patient Assistance Program for uninsured or denied patients. Pombiliti + Opfolda: Amicus Assist 1-833-AMICUS-A (1-833-264-2872), Monday to Friday 8AM to 8PM ET; Patient Referral Form by fax 1-833-264-2873; co-pay assistance for eligible commercial patients; Amicus Patient Assistance Program.
A reference for licensed professionals, checked on November 11, 2026. Labels and plan policies change; the label on DailyMed and the plan's current policy govern. Not medical advice. For patients: the Pompe disease page and the first 90 days pages for Lumizyme, Nexviazyme, Pombiliti + Opfolda.