| Glycogen storage disease type II

Pombiliti + Opfolda vs Lumizyme

Side-by-side clinical, coverage, and cost comparison for glycogen storage disease type ii.
Deep comparison between: Pombiliti vs Lumizyme with Prescriber.AI
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Safety signalsLumizyme has a higher rate of injection site reactions vs Pombiliti based on FDA-approved prescribing information
Coverage gaps3 major payers require step therapy for Lumizyme but not Pombiliti, including UnitedHealthcare
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Pombiliti
Lumizyme
At A Glance
IV infusion
Every 2 weeks
Lysosomal acid alpha-glucosidase (GAA) replacement
IV infusion
Every 2 weeks
GAA enzyme replacement
Indications
  • Glycogen storage disease type II
  • Glycogen storage disease type II
Dosing
Glycogen storage disease type II 20 mg/kg actual body weight every other week as an IV infusion over approximately 4 hours, administered in combination with Opfolda, initiated approximately 1 hour after oral Opfolda.
Glycogen storage disease type II 20 mg/kg every 2 weeks as an intravenous infusion; initial infusion rate no more than 1 mg/kg/hour, titrated by 2 mg/kg/hour every 30 minutes as tolerated to a maximum of 7 mg/kg/hour.
Contraindications
  • Pregnancy
—
Adverse Reactions
Most common (>=5%) Headache, diarrhea, fatigue, nausea, abdominal pain, pyrexia
Serious Anaphylaxis, urticaria
Most common (>=5%) anaphylaxis, rash, pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis, nausea, cough, decreased oxygen saturation, tachycardia, tachypnea, chest discomfort, dizziness, muscle twitching, agitation, cyanosis, erythema, hypertension/increased blood pressure, pallor, rigors, tremor, vomiting, fatigue, myalgia
Serious anaphylaxis, acute cardiorespiratory failure
Postmarketing anaphylactic shock, respiratory failure, respiratory arrest, cardiac arrest, hypoxia, dyspnea, wheezing, convulsions, proteinuria, nephrotic syndrome secondary to membranous glomerulonephritis, necrotizing skin lesions, hyperparathyroidism
Pharmacology
Recombinant human lysosomal acid alpha-glucosidase (rhGAA) enzyme replacement; cipaglucosidase alfa-atga binds M6P receptors via high-affinity bis-M6P moieties, is internalized into lysosomes where it undergoes proteolytic processing and cleaves accumulated glycogen; miglustat (Opfolda) stabilizes the enzyme in blood post-infusion to reduce inactivation.
Alglucosidase alfa provides an exogenous source of acid alpha-glucosidase (GAA); it binds to mannose-6-phosphate receptors on the cell surface, is internalized and transported into lysosomes where proteolytic cleavage increases enzymatic activity, and then cleaves lysosomal glycogen to address the GAA deficiency underlying Pompe disease.
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Most Common Insurance
Anthem BCBS
Pombiliti
  • Covered on 5 commercial plans
  • PA (9/12) · Step Therapy (5/12) · Qty limit (0/12)
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Lumizyme
  • Covered on 5 commercial plans
  • PA (12/12) · Step Therapy (0/12) · Qty limit (0/12)
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UnitedHealthcare
Pombiliti
  • Covered on 4 commercial plans
  • PA (0/8) · Step Therapy (0/8) · Qty limit (0/8)
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Lumizyme
  • Covered on 4 commercial plans
  • PA (0/8) · Step Therapy (0/8) · Qty limit (0/8)
View full coverage details ›
Humana
Pombiliti
  • Covered on 0 commercial plans
  • PA (2/3) · Step Therapy (0/3) · Qty limit (0/3)
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Lumizyme
  • Covered on 0 commercial plans
  • PA (2/3) · Step Therapy (0/3) · Qty limit (0/3)
View full coverage details ›
Coverage data sourced from MMIT. Updated monthly.
Savings
Cost estimate not availableAccessia Health: Pompe Disease - Private Insurance
Commercial or private insurance
Medicare, Medicaid, VA, TRICARE
Cost estimate not availableAccessia Health: Pompe Disease - Private Insurance
Commercial or private insurance
Medicare, Medicaid, VA, TRICARE
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Clinical data sourced from FDA-approved labeling. Coverage data via MMIT. Updated monthly.