By Counterforce Health Team in Myozyme (alglucosidase alfa) —

How to Get Myozyme (Alglucosidase Alfa) Covered by Aetna CVS Health in New York: Complete Prior Authorization and Appeals Guide

Answer Box: Getting Myozyme Covered by Aetna CVS Health in New York

Fastest Path to Approval: Submit prior authorization through CVS Specialty Pharmacy with confirmed Pompe disease diagnosis (GAA enzyme testing + genetic confirmation), specialist evaluation, and dosing at 20 mg/kg every 2 weeks per FDA label. If denied, file internal appeal within 65 days, then external review through New York Department of Financial Services within 4 months. Start today: Contact CVS Specialty at 1-866-814-5506 to initiate coverage verification and PA submission.

Table of Contents

Aetna CVS Health Policy Overview

Aetna CVS Health requires prior authorization for Myozyme (alglucosidase alfa) across all plan types in New York. The medication is typically handled through CVS Specialty Pharmacy, which manages most enzyme replacement therapies for rare diseases.

Coverage at a Glance

Requirement Details Source
Prior Authorization Required for all plans Aetna Precertification List
Formulary Status Specialty tier (verify current status) Contact CVS Specialty
Step Therapy May apply - exceptions available Plan-specific
Quantity Limits Based on FDA dosing (20 mg/kg q2w) FDA Label
Site of Care Infusion center or qualified facility CVS Specialty coordination
Age Restrictions FDA-approved for infantile-onset Pompe FDA Access Data

Medical Necessity Requirements

Aetna CVS Health typically requires the following documentation to establish medical necessity for Myozyme:

Primary Diagnosis Confirmation

Pompe Disease Diagnosis must be confirmed through:

  • GAA enzyme activity testing (preferred: skin fibroblasts or muscle biopsy)
  • Genetic testing confirming GAA gene mutations
  • Clinical presentation consistent with infantile-onset Pompe disease
Clinical Note: Dried blood spot (DBS) testing combined with urinary glucose tetrasaccharide analysis reaches nearly 100% diagnostic sensitivity for infantile Pompe disease.

Supporting Clinical Evidence

  • Elevated creatine kinase, AST, ALT, and LDH levels
  • Muscle biopsy showing glycogen accumulation (if performed)
  • Echocardiogram findings (cardiomyopathy in infantile cases)
  • Respiratory function assessments

Specialist Evaluation

Treatment must be prescribed by or in consultation with a physician experienced in Pompe disease management, typically:

  • Geneticist
  • Neurologist specializing in neuromuscular disorders
  • Metabolic specialist

Step Therapy and Exceptions

Standard Step Therapy

For infantile-onset Pompe disease, Myozyme is typically first-line therapy as it's the original FDA-approved enzyme replacement therapy. However, some plans may require consideration of:

  • Lumizyme (alglucosidase alfa) - broader Pompe indications
  • Documentation of why Myozyme specifically is preferred

Medical Exception Pathways

Step therapy exceptions are typically granted when:

  • Patient has contraindications to alternative therapies
  • Previous treatment failures with other ERTs
  • Specialist recommendation for Myozyme specifically
  • Urgent clinical need (cardiomyopathy, respiratory compromise)

Dosing and Quantity Limits

FDA-Approved Dosing

Standard Dose: 20 mg/kg body weight administered intravenously every 2 weeks

Weight-Based Calculations

Myozyme is supplied in 50-mg single-use vials. Coverage calculations are based on:

  • Patient's actual body weight in kilograms
  • Biweekly administration schedule
  • Appropriate vial quantities to minimize waste
Example: A 10 kg infant requires 200 mg per dose (4 vials) every 2 weeks.

Higher Dose Considerations

While FDA labeling specifies 20 mg/kg, clinical evidence suggests approximately 52% of patients may benefit from higher doses. Higher doses require additional documentation:

  • Clinical rationale for dose escalation
  • Treatment response monitoring
  • Specialist recommendation

Required Diagnostics

Laboratory Tests

Required for Initial Authorization:

  • GAA enzyme activity levels
  • Creatine kinase (CK) levels
  • Liver function tests (AST, ALT)
  • LDH levels
  • Urinary glucose tetrasaccharide (if available)

Timing Requirements:

  • Diagnostic tests should be recent (typically within 6 months)
  • Baseline cardiac and respiratory function assessments

Genetic Testing

  • GAA gene sequencing confirming pathogenic mutations
  • Family history documentation (autosomal recessive inheritance)
  • Genetic counseling documentation

Site of Care Requirements

Approved Administration Settings

Myozyme must be administered in facilities equipped to manage infusion reactions:

  • Hospital-based infusion centers
  • Specialty infusion clinics
  • CVS Specialty infusion suites (where available)

Safety Requirements

Given the risk of anaphylaxis and infusion reactions, administration sites must have:

  • Emergency resuscitation equipment
  • Trained healthcare professionals
  • Protocols for managing allergic reactions

CVS Specialty Pharmacy offers flexible site of care options for infusion therapies, but specific requirements should be verified for Myozyme.

Evidence to Support Approval

Medical Necessity Letter Checklist

For Clinicians: Your prior authorization letter should include:

  1. Problem Statement
    • Confirmed Pompe disease diagnosis
    • Current clinical status and symptoms
    • Disease progression without treatment
  2. Prior Treatments
    • Supportive care measures tried
    • Any previous enzyme replacement therapy
    • Treatment outcomes and limitations
  3. Clinical Rationale
    • Why Myozyme is medically necessary
    • Expected treatment benefits
    • Monitoring plan
  4. Guideline Support
    • FDA approval for infantile-onset Pompe disease
    • Published treatment guidelines
    • Specialist society recommendations

Appeals Process in New York

Internal Appeals with Aetna CVS Health

Timeline: 65 days from denial notice to file appeal

Decision Timeline:

  • Standard review: 7 days
  • Expedited review: 72 hours (for urgent needs)

How to File:

  • Online through Aetna member portal
  • Fax: 1-855-633-7673
  • Phone: 1-866-235-5660

External Review Through New York DFS

When to Use: After receiving final internal denial from Aetna

Timeline: 4 months from internal appeal decision to file external review

Decision Timeline:

  • Standard: Up to 30 calendar days
  • Expedited: 72 hours for urgent medical needs

Contact: New York Department of Financial Services External Appeal Program

New York Advantage: The state's external appeal process is binding on insurers and has successfully overturned many specialty drug denials when medical necessity is clearly documented.

Appeals Support

Free Assistance: Community Health Advocates provides free counseling for New York residents at 888-614-5400.

Common Denial Reasons and Solutions

Denial Reason Solution Documentation Needed
"Not medically necessary" Provide specialist evaluation and clinical evidence GAA enzyme levels, genetic testing, clinical progression
"Experimental/investigational" Cite FDA approval for infantile-onset Pompe FDA label, approval date, indication
"Non-formulary" Request formulary exception Medical necessity letter, no suitable alternatives
"Dosing exceeds guidelines" Provide clinical rationale for dose Specialist recommendation, treatment response data
"Inadequate diagnosis" Submit complete diagnostic workup Enzyme testing, genetic confirmation, specialist evaluation

Counterforce Health specializes in turning insurance denials into targeted, evidence-backed appeals. Their platform helps identify the specific denial basis and drafts point-by-point rebuttals aligned to each plan's own rules, significantly improving approval rates for complex specialty medications like Myozyme.

Cost Assistance Programs

Manufacturer Support

Sanofi Patient Assistance: Contact Sanofi directly for patient assistance programs and copay support options.

Foundation Grants

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

New York State Programs

For Medicaid enrollees, coverage is typically more comprehensive, though prior authorization may still be required.

FAQ

How long does Aetna CVS Health prior authorization take for Myozyme in New York? Standard reviews are completed within 72 hours, with expedited reviews available within 24 hours for urgent medical needs.

What if Myozyme is non-formulary on my Aetna plan? Request a formulary exception with medical necessity documentation. Include evidence that no suitable formulary alternatives exist for infantile-onset Pompe disease.

Can I request an expedited appeal for Myozyme denial? Yes, if delayed treatment poses serious health risks. Both Aetna's internal appeals and New York's external review offer expedited pathways with 72-hour decisions.

Does step therapy apply if I've tried treatments outside New York? Document all prior treatments regardless of location. Out-of-state treatment failures should be accepted if properly documented.

What happens if I miss the appeal deadline? Contact New York Department of Financial Services immediately. Some exceptions may be available for good cause, but it's critical to file within required timeframes.

How much does Myozyme cost without insurance? Cash-pay costs approximately $1,026 per 50-mg vial, with treatment requiring multiple vials per dose based on patient weight.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical advice. Coverage policies vary by individual plan and change frequently. Always verify current requirements with your insurance provider and consult your healthcare team for medical decisions. For personalized assistance with insurance appeals and prior authorization, visit Counterforce Health to learn how their platform helps patients and clinicians navigate complex coverage requirements for specialty medications.

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