By Counterforce Health Team in Pombiliti (cipaglucosidase alfa-atga) —

The Requirements Checklist to Get Pombiliti (cipaglucosidase alfa-atga) Covered by Aetna (CVS Health) in Pennsylvania

Answer Box: Your Fastest Path to Coverage

Getting Pombiliti (cipaglucosidase alfa-atga) covered by Aetna (CVS Health) in Pennsylvania requires meeting specific clinical criteria and proper documentation. You must be 18+ years old, weigh ≥40 kg, have confirmed late-onset Pompe disease, and demonstrate inadequate response to current enzyme replacement therapy. Start today by: 1) Gathering your GAA enzyme test and genetic confirmation, 2) Documenting prior ERT failures, and 3) Having your specialist submit a prior authorization through Aetna's provider portal. Appeals in Pennsylvania have strong success rates—about 50% of external reviews overturn denials.

Table of Contents

  1. Who Should Use This Checklist
  2. Member & Plan Basics
  3. Clinical Criteria Requirements
  4. Coding and Billing Information
  5. Documentation Packet
  6. Submission Process
  7. Specialty Pharmacy Requirements
  8. After Submission: What to Expect
  9. Common Denial Reasons & How to Fix Them
  10. Pennsylvania Appeals Process
  11. Printable Checklist

Who Should Use This Checklist

This guide is designed for patients with late-onset Pompe disease (LOPD) and their healthcare teams seeking Aetna (CVS Health) coverage for Pombiliti in Pennsylvania. You'll benefit most if you:

  • Have confirmed LOPD diagnosis with enzyme and genetic testing
  • Are currently on enzyme replacement therapy (ERT) but not improving adequately
  • Need to switch from Lumizyme or Nexviazyme to the Pombiliti + Opfolda combination
  • Face a prior authorization denial and need to appeal

Expected outcome: With proper documentation meeting Aetna's clinical criteria, most requests are approved. If denied initially, Pennsylvania's external review process has overturned about 50% of insurance denials in its first year.

Member & Plan Basics

Active Coverage Requirements

  • Valid Aetna membership in Pennsylvania
  • Specialty drug benefits included in your plan
  • Current deductible status (verify if met for the year)

Plan Types That Cover Pombiliti

  • Commercial employer plans
  • Individual marketplace plans (including Pennie purchases)
  • Medicare Advantage plans (different criteria may apply)
Tip: Call the member services number on your insurance card to confirm your specialty drug coverage and any applicable copays before starting the prior authorization process.

Clinical Criteria Requirements

Based on Aetna's Medical Clinical Policy #0442, you must meet all of the following:

Patient Demographics

  • Age: 18 years or older
  • Weight: ≥40 kg (88 pounds)

Diagnosis Confirmation

  • Late-onset Pompe disease confirmed by:
    • GAA enzyme activity assay showing deficiency, OR
    • Genetic testing confirming pathogenic GAA mutations

Step Therapy Requirement

  • Patient must not be improving on current ERT (Lumizyme or Nexviazyme)
  • Documentation of inadequate response required

Combination Therapy

  • Pombiliti must be prescribed with Opfolda (miglustat)
  • Cannot be used with other GAA enzyme replacement therapies

Prescriber Requirements

  • Prescribed by or in consultation with a metabolic or lysosomal storage disorder specialist

Coding and Billing Information

ICD-10 Diagnosis Code

  • E74.02 - Glycogen storage disease type II (specify "late-onset" in clinical notes)

Drug Information

  • Generic name: cipaglucosidase alfa-atga
  • Dosing: 20 mg/kg IV every 2 weeks over ~4 hours
  • NDC numbers: (verify current NDCs with CVS Specialty)
  • J-code: Not yet assigned (verify with billing department)

Companion Drug

  • Opfolda (miglustat) must be taken ~1 hour before each Pombiliti infusion

Documentation Packet

Provider Clinical Note Must Include:

  1. Diagnosis confirmation:
    • GAA enzyme activity results
    • Genetic testing results showing pathogenic mutations
    • ICD-10 code E74.02
  2. Prior therapy documentation:
    • Current or recent ERT (Lumizyme/Nexviazyme)
    • Evidence of inadequate response (functional assessments, symptom progression)
    • Duration of prior treatment
  3. Clinical justification:
    • Current symptoms and functional status
    • Treatment goals with Pombiliti + Opfolda
    • Monitoring plan

Letter of Medical Necessity Components

Your specialist should include:

  • Patient demographics and diagnosis
  • Clinical history and prior treatments
  • Rationale for Pombiliti + Opfolda combination
  • References to FDA labeling and clinical guidelines
  • Expected outcomes and monitoring plan

Required Attachments

  • Lab reports (GAA enzyme activity)
  • Genetic testing results
  • Recent clinic notes documenting ERT response
  • Functional assessment scores (if available)

Submission Process

Prior Authorization Submission

  1. Complete Aetna's specialty drug PA form (current version available through provider portal)
  2. Submit via: Aetna provider portal or designated fax line
  3. Include all required fields: Patient demographics, diagnosis codes, drug information, clinical justification

Required Fields That Commonly Cause Rejections

  • Incomplete patient weight documentation
  • Missing GAA enzyme or genetic test results
  • Vague description of prior ERT response
  • Incorrect ICD-10 coding
  • Missing specialist involvement documentation

Timeline

  • Standard review: 15-30 days
  • Expedited review: 72 hours (for urgent cases)

Specialty Pharmacy Requirements

Aetna requires Pombiliti to be dispensed through CVS Specialty Pharmacy or another contracted specialty vendor.

Transfer Process

  1. Prescription routing: Your doctor submits the prescription directly to CVS Specialty
  2. Benefits verification: CVS Specialty confirms coverage and copay
  3. Shipment coordination: Temperature-controlled delivery to infusion site
  4. Opfolda coordination: Ensure oral medication is also ordered through specialty pharmacy

CVS Specialty Contact

  • Patients can track orders and coordinate delivery through CVS Specialty's patient portal
  • Providers coordinate through the CVS Specialty provider portal

After Submission: What to Expect

Confirmation and Tracking

  • Record your confirmation number from the PA submission
  • Check status weekly through the provider portal
  • Expected timeline: 15-30 days for standard review

Possible Outcomes

  • Approved: Coverage confirmed, proceed with CVS Specialty
  • Denied: Review denial letter for specific reasons
  • More information needed: Respond promptly to requests
Note: Keep detailed records of all submissions, confirmation numbers, and communications for potential appeals.

Common Denial Reasons & How to Fix Them

Denial Reason How to Fix Required Documentation
Diagnosis not confirmed Submit complete diagnostic workup GAA enzyme results + genetic testing
Inadequate prior ERT trial Document treatment history and response Clinic notes showing lack of improvement
Missing specialist involvement Get specialist consultation Letter from metabolic specialist
Incomplete clinical information Provide comprehensive medical history Detailed provider notes with ICD-10
Step therapy not met Demonstrate ERT failure/intolerance Functional assessments, side effect documentation

Pennsylvania Appeals Process

Pennsylvania offers robust appeal rights with strong success rates. Counterforce Health helps patients and clinicians turn insurance denials into targeted, evidence-backed appeals by analyzing denial letters and crafting point-by-point rebuttals aligned with payer policies.

Internal Appeals (First Step)

  • Timeline: Must file within 180 days of denial
  • Process: Submit through Aetna member portal or by mail
  • Decision time: 30-45 days (72 hours for urgent)

External Review (If Internal Appeal Fails)

Pennsylvania's Independent External Review Program has overturned about 50% of denials in its first year.

Key Details:

  • Eligibility: Must complete internal appeals first
  • Timeline: 4 months to file after final denial
  • Decision time: 45 days standard, 72 hours expedited
  • Success rate: ~50% overturn rate statewide

Required Documentation for Appeals

  • Final denial letter from Aetna
  • All medical records supporting medical necessity
  • Specialist letters explaining clinical rationale
  • Research citations supporting off-label use (if applicable)
From our advocates: We've seen successful appeals when patients include detailed functional assessments showing decline on current ERT, combined with peer-reviewed studies demonstrating Pombiliti's superior tissue uptake. The key is connecting your specific clinical picture to the scientific evidence base.

Printable Checklist

Before You Start: □ Active Aetna coverage confirmed
□ Patient ≥18 years old and ≥40 kg
□ LOPD diagnosis confirmed (enzyme + genetic testing)
□ Currently on ERT with inadequate response documented
□ Metabolic specialist involved in care

Documentation Gathered: □ GAA enzyme activity results
□ Genetic testing confirming pathogenic mutations
□ Recent clinic notes with ICD-10 E74.02
□ Prior ERT treatment history and response
□ Functional assessment scores (if available)

Submission Complete: □ PA form submitted via Aetna provider portal
□ All required fields completed accurately
□ Confirmation number recorded
□ CVS Specialty pharmacy contacted
□ Opfolda prescription coordinated

After Submission: □ Status checked weekly
□ Response timeline tracked (15-30 days)
□ Appeal strategy prepared if needed
□ Pennsylvania external review process understood

Frequently Asked Questions

How long does Aetna prior authorization take in Pennsylvania? Standard reviews take 15-30 days. Expedited reviews for urgent cases are completed within 72 hours.

What if Pombiliti is not on Aetna's formulary? You can request a formulary exception with documentation of medical necessity and prior ERT failure.

Can I get an expedited appeal if denied? Yes, if your doctor certifies that the delay poses a serious risk to your health or life, you can request expedited review with 72-hour decision timeline.

Does step therapy apply if I've tried ERT outside Pennsylvania? Yes, prior ERT trials from any location count toward meeting Aetna's step therapy requirements.

What copay should I expect? Specialty drug copays vary by plan, typically ranging from $0-$100 per month, but high-cost drugs may have coinsurance instead.

Cost Assistance Options

Manufacturer Support

  • Amicus Assist: Patient support program offering copay assistance and prior authorization help
  • Eligibility: Commercial insurance patients may qualify for copay reduction

Foundation Grants

  • Search rare disease foundations for Pompe-specific assistance programs
  • Many foundations offer grants for patients with high out-of-pocket costs

Counterforce Health helps patients, clinicians, and specialty pharmacies get prescription drugs approved by turning insurance denials into targeted, evidence-backed appeals. The platform analyzes denial letters, plan policies, and clinical notes to identify the denial basis and draft point-by-point rebuttals aligned to the plan's own rules. For Pombiliti appeals, Counterforce Health can help weave together the right citations—FDA labeling, peer-reviewed studies, and specialty guidelines—with required clinical facts like diagnosis codes, prior ERT failures, and treatment goals.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Always consult with your healthcare provider and insurance plan for specific coverage decisions. Requirements and processes may change—verify current information with official sources before making healthcare decisions.

Powered by Counterforce Health—AI that turns drug denials into evidence-based appeals patients and clinicians can submit today.