By Counterforce Health Team in Myozyme (alglucosidase alfa) —

How to Get Myozyme (Alglucosidase Alfa) Covered by Cigna in North Carolina: Complete Prior Authorization and Appeals Guide

Answer Box: Fastest Path to Myozyme Coverage

To get Myozyme (alglucosidase alfa) covered by Cigna in North Carolina, you need: (1) confirmed Pompe disease diagnosis with GAA enzyme testing and/or genetic results, (2) prescription from a metabolic/neuromuscular specialist, and (3) prior authorization submission via Cigna's provider portal or specialty pharmacy. If denied, complete internal appeals within 180 days, then request external review through Smart NC. Standard PA approval takes 72 hours to 14 days; expedited reviews available for urgent cases.

Table of Contents

Cigna's Coverage Policy for Myozyme

Cigna covers Myozyme (alglucosidase alfa) as a specialty medication for confirmed Pompe disease across commercial, Medicare Advantage, and Medicaid plans in North Carolina. Coverage requires prior authorization and typically routes through Express Scripts/Accredo specialty pharmacy.

Plan Types and Coverage Differences

Commercial Plans: Follow Cigna's IP-0440 coverage policy requiring specialist prescriber and confirmed diagnosis. Internal appeals have 180-day filing window.

Medicare Advantage/Part D: Subject to Medicare PA timelines (65 days for first appeal). Coverage determination process follows CMS guidelines with potential for 5 appeal levels.

Medicaid Plans: North Carolina Medicaid expansion (effective 2024) may provide additional coverage pathways for eligible patients.

Note: Self-funded employer plans may have limited external review rights in North Carolina. Contact Smart NC at 1-855-408-1212 to verify your appeal rights.

Medical Necessity Requirements

Cigna requires objective confirmation of Pompe disease and specialist oversight for Myozyme coverage. The drug is FDA-approved for both infantile-onset and late-onset Pompe disease.

Coverage at a Glance

Requirement What It Means Documentation Needed Source
Confirmed Diagnosis Pompe disease (ICD-10: E74.02) GAA enzyme assay + genetic testing FDA Label
Specialist Prescriber Metabolic, genetic, or neuromuscular specialist Prescriber credentials/board certification Cigna PA Policy
Prior Authorization Required for all plans Completed PA form + clinical documentation Cigna PA Forms
Dosing Compliance 20 mg/kg IV every 2 weeks Weight-based calculation documentation FDA Label

Step Therapy and Exceptions

Good news: Cigna does not require step therapy for Myozyme in confirmed Pompe disease cases. There are no clinically appropriate alternatives that patients must try first.

Medical Exception Pathways

If your plan initially requires step therapy (rare but possible), you can request an exception by documenting:

  • Lack of therapeutic alternatives for enzyme replacement in Pompe disease
  • Disease severity requiring immediate treatment
  • Contraindications to any suggested alternative therapies

Submit exception requests using Cigna's formulary exception process with specialist support.

Dosing and Quantity Limits

Cigna typically approves Myozyme at FDA-labeled dosing: 20 mg/kg IV every 2 weeks. Higher doses (40 mg/kg) require additional medical justification.

Standard Coverage Parameters

  • Maximum dose: 20 mg/kg every 14 days
  • Annual limit: 26 infusions per year
  • Vial calculations: Based on current weight and 50-mg vial size
  • Renewal requirements: Updated weight, clinical response documentation

Dose Adjustment Process

When weight changes require dose modifications:

  1. Document new weight with measurement date
  2. Recalculate total mg dose (weight × 20 mg/kg)
  3. Submit updated PA with revised vial count
  4. Provide clinical justification for any dose increases
Tip: Pediatric patients may need frequent dose adjustments as they grow. Plan for quarterly PA updates to avoid coverage gaps.

Required Diagnostic Documentation

Strong diagnostic documentation is crucial for Myozyme approval. Cigna requires objective proof of Pompe disease through laboratory testing.

Diagnostic Testing Pathway

First-Line Testing:

  • GAA enzyme activity via dried blood spot (DBS) or leukocyte assay
  • Elevated creatine/creatinine ratio (supportive evidence)
  • Urine glucotetrasaccharide (Glc4) levels

Confirmatory Testing:

  • GAA gene sequencing identifying pathogenic variants
  • Two disease-causing mutations (autosomal recessive inheritance)
  • Genetic counseling documentation

Documentation Checklist

  • Laboratory reports showing reduced GAA activity
  • Genetic test results with pathogenic variant identification
  • Clinical notes describing symptoms and disease progression
  • Specialist evaluation confirming Pompe diagnosis
  • Family history if applicable
  • Cardiac/pulmonary function studies showing disease impact

Specialty Pharmacy Requirements

Most Cigna plans require Myozyme to be dispensed through Accredo specialty pharmacy rather than retail pharmacies.

Accredo Enrollment Process

  1. Prescriber enrollment: Complete Accredo provider registration
  2. Patient enrollment: Create MyAccredoPatients account
  3. Benefits verification: Accredo confirms PA status and coverage
  4. Delivery coordination: Schedule shipments to infusion site

Site of Care Considerations

Approved Infusion Settings:

  • Hospital outpatient infusion centers
  • Specialty infusion clinics
  • Home infusion (with appropriate monitoring)

Network Requirements:

  • Verify infusion provider is in Cigna's medical network
  • Confirm site can receive Accredo deliveries
  • Ensure emergency management capabilities for infusion reactions
Important: Using out-of-network specialty pharmacies can result in denial or significantly higher out-of-pocket costs.

Evidence to Support Your Case

When submitting PA requests or appeals, include evidence-based support for Myozyme therapy in Pompe disease.

Key Clinical Guidelines

  • FDA prescribing information establishing efficacy and safety
  • Pompe disease management guidelines from specialty societies
  • Published clinical trials demonstrating improved outcomes
  • Real-world evidence of functional improvements

Medical Necessity Letter Components

Your specialist should address:

  1. Specific Pompe disease subtype (infantile vs. late-onset)
  2. Disease progression and current functional status
  3. Expected benefits of enzyme replacement therapy
  4. Lack of alternative treatments
  5. Monitoring plan for safety and efficacy
  6. Dosing rationale based on current weight

Appeals Process in North Carolina

If Cigna denies your Myozyme request, North Carolina offers strong consumer protections through internal appeals and external review.

Internal Appeals Timeline

Commercial Plans:

  • Filing deadline: 180 days from denial date
  • Review timeline: 30 days for medical necessity appeals
  • Expedited review: Available for urgent situations

Medicare Advantage:

  • Level 1 (Redetermination): 65 days to file, 7 days for decision
  • Level 2 (Independent Review): 60 days to file, 30 days for decision

Smart NC External Review

After exhausting internal appeals, North Carolina residents can request independent external review through Smart NC.

Eligibility Requirements:

  • Completed Cigna internal appeals process
  • State-regulated insurance plan (not self-funded ERISA plans)
  • Denial based on medical necessity or experimental determination

Process Overview:

  1. Contact Smart NC at 1-855-408-1212 for eligibility verification
  2. Submit external review request with all documentation
  3. Independent Review Organization (IRO) conducts clinical review
  4. Binding decision within 45 days (72 hours for expedited)
Key Advantage: North Carolina's external review has helped overturn many specialty drug denials, especially for rare diseases with strong clinical evidence.

Common Denial Reasons and Solutions

Denial Reason How to Overturn Documentation Needed
"Experimental/Investigational" Cite FDA approval and clinical guidelines FDA label, published studies, specialist letter
"Not Medically Necessary" Document disease severity and lack of alternatives Clinical notes, functional assessments, progression data
"Insufficient Documentation" Provide complete diagnostic workup All lab results, genetic testing, specialist evaluation
"Non-Formulary Drug" Request formulary exception Medical necessity letter, guideline citations
"Incorrect Prescriber" Transfer to qualified specialist Specialist credentials, board certification
"Dosing Outside Guidelines" Justify weight-based calculations Current weight, dosing calculations, FDA label

Appeal Strategy Tips

  • Address every denial reason point-by-point in your appeal letter
  • Include peer-reviewed evidence supporting Myozyme use in Pompe disease
  • Request expedited review if delays could worsen health outcomes
  • Consider peer-to-peer review between your specialist and Cigna medical director

Costs and Patient Assistance

Myozyme is expensive (approximately $1,026 per 50-mg vial), but multiple assistance programs can help reduce out-of-pocket costs.

Manufacturer Support

Sanofi Patient Assistance:

  • Copay assistance for commercially insured patients
  • Free drug program for uninsured/underinsured patients
  • Income-based eligibility requirements

Foundation Grants

  • National Organization for Rare Disorders (NORD)
  • Patient Access Network Foundation
  • Good Days (formerly Chronic Disease Fund)

State Programs

North Carolina's Medicaid expansion may provide coverage for newly eligible patients. Contact NC Medicaid for eligibility screening.

FAQ

How long does Cigna prior authorization take for Myozyme in North Carolina?

Standard PA decisions take 72 hours to 14 days. Expedited reviews for urgent cases are completed within 24-72 hours. Submit complete documentation to avoid delays.

What if Myozyme is non-formulary on my Cigna plan?

Request a formulary exception through Cigna's non-covered medication process. Include medical necessity documentation and specialist support for the request.

Can I request an expedited appeal if Cigna denies Myozyme?

Yes, expedited appeals are available when delays could seriously jeopardize your health. This is particularly relevant for infantile-onset Pompe disease or rapidly progressing cases.

Does step therapy apply if I've tried treatments outside North Carolina?

Document all prior treatments, including those received in other states. Cigna generally does not require step therapy for Myozyme in confirmed Pompe disease.

What happens if Smart NC external review upholds the denial?

External review decisions are typically final for state-regulated plans. You may still have legal rights to pursue, but administrative appeals are exhausted.

How often do I need to renew Myozyme prior authorization?

Most approvals are granted for one year. Renewals require updated clinical documentation showing continued medical necessity and appropriate response to treatment.

Counterforce Health specializes in turning insurance denials into evidence-backed appeals for complex medications like Myozyme. Our platform helps patients, clinicians, and specialty pharmacies navigate prior authorization requirements and craft targeted appeals that address payer-specific criteria. By analyzing denial letters alongside plan policies and clinical notes, Counterforce Health streamlines the appeals process and improves approval rates for life-saving therapies.

When facing a Myozyme denial, remember that North Carolina offers robust consumer protections through Smart NC's external review process. Many specialty drug denials are overturned when complete clinical documentation is provided to independent medical reviewers. Counterforce Health's evidence-based approach helps ensure your appeal includes all the clinical facts and guideline citations payers need to approve coverage.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Insurance coverage decisions depend on individual plan terms and clinical circumstances. Always consult with your healthcare provider and insurance company for guidance specific to your situation. For assistance with North Carolina insurance appeals, contact Smart NC at 1-855-408-1212.

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