CommercialPrior AuthHigh impact
Lysosomal Storage Disorder Treatments (CPB 0442, reviewed 2026-07-08)
Aetna·Genetics, Internal Medicine, Pediatrics +3 more·Medical Policy
Effective date
Jul 8, 2026
We identified it
Aug 16, 2026
Summary
Aetna has updated its Lysosomal Storage Disorder treatment policy (CPB 0442, effective 2026-07-08) establishing cost-based medical necessity criteria for enzyme replacement therapies. For Gaucher Disease, VPRIV is now limited to members who fail or are intolerant to Cerezyme and Elelyso. For Pompe Disease, Lumizyme and Pombiliti are restricted to members failing Nexviazyme therapy. All enzyme replacement drugs now require precertification, and treatment must be prescribed by specialists in metabolic disease or lysosomal storage disorders.
Action Required
By 2026-07-08, billing and clinical teams must: (1) Update authorization workflows to implement step-therapy requirements—Gaucher Disease patients must document failure/intolerance to Cerezyme AND Elelyso before approving VPRIV claims; Pompe Disease patients must document failure/intolerance to Nexviazyme before approving Lumizyme or Pombiliti. (2) Implement mandatory precertification for all enzyme replacement drug claims by calling (866) 752-7021 or faxing (888) 267-3277 before dispensing; obtain Statement of Medical Necessity (SMN) forms via Specialty Pharmacy Precertification portal. (3) Update claim denial templates to reject requests for VPRIV, Lumizyme, and Pombiliti that do not document step-therapy failure. (4) Verify that prescribers are credentialed specialists in metabolic disease or lysosomal storage disorders; flag non-specialist prescriptions for peer-to-peer review. (5) Educate providers and patients that claims without proper step-therapy documentation and precertification will be denied. (6) Train specialty pharmacy staff on site-of-care utilization management requirements for all listed enzyme replacement drugs (Aldurazyme, Brineura, Cerezyme, Elaprase, Elelyso, Elfabrio, Fabrazyme, Kanuma, Lamzede, Lumizyme, Mepsevii, Naglazyme, Nexviazyme, Pombiliti, Vimizim, Vpriv, and Xenpozyme).