US Pompe ERT pricing runs almost entirely through Medicare Part B, Pombiliti + Opfolda alone splits across two benefit categories, and no ICER review exists to anchor a value-based price.
All three approved US Pompe enzyme replacement therapies are intravenous infusions billed under the Medicare Part B medical benefit, which removes patient out-of-pocket cost for most Medicare and Medicaid beneficiaries. Alglucosidase alfa (Lumizyme/Myozyme) carries an annual WAC near $400,000, and avalglucosidase alfa (Nexviazyme) prices at roughly $400,000 to $450,000, a modest premium tied to its COMET six-minute-walk advantage. The Pombiliti + Opfolda regimen breaks this pattern: cipaglucosidase alfa infuses under Part B while its oral chaperone, miglustat, routes to Part D, creating a $600,000 to $800,000 combined annual cost and a two-benefit coordination burden that some plans are still not configured to process cleanly.
Payers treat alglucosidase alfa as the step-edit incumbent regardless of trial data: most commercial plans require six to twelve months of documented inadequate response before authorizing a next-generation switch, a friction point that shapes launch sequencing more than any efficacy argument. Pompe disease has never received a formal ICER cost-effectiveness review, unlike myasthenia gravis, SMA, or ATTR amyloidosis; a review is expected in 2025, and until it lands payers benchmark against Gaucher-disease ERT rather than a Pompe-specific QALY threshold. A new entrant should plan for a 12-month HCPCS J-code lead time and expect its price ladder to be judged against the $400,000 to $800,000 band already established.
US Pompe ERT pricing — WAC and benefit-routing compared across all three approved agents
| Agent | Benefit Routing | Annual WAC (est.) | Key Access Barrier |
|---|---|---|---|
| Alglucosidase alfa (Lumizyme/Myozyme) | Medicare Part B (IV infusion) | ~$400,000/yr | Step-edit incumbent; easiest PA, longest track record |
| Avalglucosidase alfa (Nexviazyme) | Medicare Part B (IV infusion) | ~$400,000-450,000/yr | Some plans require documented alglucosidase inadequate response first |
| Cipaglucosidase alfa + miglustat (Pombiliti + Opfolda) | Split: Part B (infusion) + Part D (oral chaperone) | ~$600,000-800,000/yr combined | Dual simultaneous prior authorization across two benefit categories |
Sources: Sanofi / Amicus Therapeutics pricing disclosures 2023; CMS HCPCS 2023 (Pombiliti / Opfolda benefit routing); ICER Evidence Report database 2024 (Pompe absence confirmed); CVS Caremark and OptumRx Pompe ERT prior-authorization policy 2024.
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- The Part B/Part D benefit-routing mechanism
- the dual simultaneous prior-authorization burden it creates
- which plans are and are not configured to process it
Delivers
- WAC benchmarks across alglucosidase alfa, avalglucosidase alfa, and Pombiliti + Opfolda
- the step-edit friction that shapes adoption
- the 12-month J-code lead time
Delivers
- Why Pompe has no ICER cost-effectiveness review yet, the 2025 review timeline, and how payers currently benchmark using the Gaucher-disease ERT analogy instead
Custom model delivered in 72 hours.
Commission This ModelWhat's inside
- Why the Part B/Part D split-benefit structure, not the molecule itself, is the binding constraint on Pombiliti + Opfolda uptake
- Pressure-tested against the zero-ICER-review gap before the rest of the model is built out
- Alglucosidase alfa, avalglucosidase alfa, and Pombiliti + Opfolda WAC compared
- Where the $600,000-800,000 combined Pombiliti + Opfolda cost sits against single-product ERT
- How the infusion and oral-chaperone components route to separate benefit categories
- The dual prior-authorization burden and which plans are configured to process it
- The 6-12 month documented-inadequate-response requirement most commercial plans apply
- What a new entrant needs in hand to clear it
- Why Pompe has zero ICER reviews to date and one expected in 2025
- How payers currently benchmark using the Gaucher-disease ERT analogy
- ASP+6% Part B reimbursement mechanics and the GTN components specific to a J-code launch
- The 12-month J-code lead time and Q-code bridging period
- Sequencing recommendation against the established $400,000-800,000 competitive band
- Conservative, base, and aggressive revenue scenarios tied to the 2025 ICER review timeline
- The open pricing questions your team must close before the US launch price is locked
- Structured for an internal pricing committee session
Included with every brief
How AXLRx builds this model
Prepared by MoatRx analysts.
Every AXLRx pricing model is built from primary regulatory and payer sources, not secondary summaries. Findings are independently verified before inclusion.
Pompe US pricing sources: Sanofi and Amicus Therapeutics pricing disclosures 2023, CMS HCPCS Part B/Part D benefit-routing documentation, the ICER Evidence Report database (Pompe absence confirmed, 2024), and CVS Caremark/OptumRx specialty-pharmacy prior-authorization policy.
- WAC figures for all three agents verified against manufacturer pricing disclosures 2023
- Part B/Part D split-benefit routing verified against CMS HCPCS 2023 documentation
- Absence of an ICER Pompe review verified against the ICER Evidence Report database 2024
Frequently asked questions
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