By Counterforce Health Team in Exondys 51 (eteplirsen) —

How to Get Exondys 51 (Eteplirsen) Covered by Aetna CVS Health in California: Prior Authorization Guide and Appeals Process

Answer Box: Fast Track to Approval

To get Exondys 51 (eteplirsen) covered by Aetna CVS Health in California: Submit prior authorization with genetic testing confirming exon 51 amenable DMD mutation, specialist prescription, baseline functional assessments (6-minute walk test ≥300m), and corticosteroid trial documentation. If denied, file internal appeal within 21 days, then request California Independent Medical Review (IMR) through DMHC for binding external review. Start today: Contact your neurologist to order genetic testing and schedule baseline assessments while gathering prior therapy records.

Table of Contents

  1. Coverage Requirements at a Glance
  2. Step-by-Step: Fastest Path to Approval
  3. When Alternatives Make Sense
  4. Common Denial Reasons & Fixes
  5. Appeals Process in California
  6. Switching Between DMD Treatments
  7. Cost Support and Patient Assistance
  8. FAQ

Coverage Requirements at a Glance

Requirement What It Means Where to Find It
Genetic Testing Confirmed DMD gene mutation amenable to exon 51 skipping Lab report from certified genetics lab
Specialist Prescriber Board-certified neurologist or neuromuscular specialist Provider credentials verification
Baseline Assessments 6MWT ≥300m independently OR NSAA >17 Functional testing at DMD clinic
Corticosteroid Trial ≥6 months standard therapy with adherence evidence Pharmacy records and clinic notes
Age Criteria Typically initiated before age 14 (ambulatory patients) Medical records documentation
Dosing Limits ≤30 mg/kg weekly IV infusion FDA prescribing information

Sources: Based on typical commercial payer criteria similar to Illinois Medicaid and UnitedHealthcare policies

Step-by-Step: Fastest Path to Approval

1. Confirm Genetic Eligibility

Who: Patient/family with neurologist
Document needed: Genetic test confirming exon 51 amenable mutation
Timeline: 2-4 weeks for results
Submit to: Include with PA submission

2. Gather Baseline Assessments

Who: DMD specialist clinic
Documents needed:

  • 6-minute walk test (≥300 meters)
  • North Star Ambulatory Assessment (NSAA >17)
  • Baseline labs (BUN, creatinine, urinalysis)
  • Cardiac function assessment Timeline: 1-2 clinic visits

3. Document Prior Therapies

Who: Clinic staff
Evidence needed: ≥6 months corticosteroid therapy (deflazacort or prednisone) with adherence records or documented intolerance
Source: Pharmacy fill history and clinic notes

4. Submit Prior Authorization

Who: Prescribing physician
How: Aetna provider portal or CVS Caremark specialty pharmacy
Timeline: Standard decision within 30-45 days; expedited ≤72 hours if urgent
Include: All baseline assessments, genetic report, specialist consultation notes

Tip: Submit PA 60-90 days before planned treatment start to allow time for appeals if initially denied.

5. Follow Up on Decision

Who: Patient or clinic
Contact: Aetna member services (number on ID card)
Timeline: Call if no response within stated timeframe

When Alternatives Make Sense

If Exondys 51 isn't immediately approved, several factors determine whether to pursue alternatives or continue fighting for coverage:

Genetic Compatibility

Other exon-skipping therapies require different mutations:

  • Amondys 45 (casimersen): Exon 45 amenable mutations (~8-13% of DMD patients)
  • Vyondys 53 (golodirsen): Exon 53 amenable mutations (~8% of patients)
  • Viltepso (viltolarsen): Also targets exon 53

Broad-Spectrum Options

Corticosteroids work regardless of genetic subtype:

  • Deflazacort (Emflaza): Often preferred first-line, fewer side effects than prednisone
  • Prednisone: Generic option, extensive long-term data
  • Vamorolone (Agamree): Newer option with potentially reduced side effects

Emerging Therapies

  • Gene therapy (Elevidys): For ambulatory patients ages 4-5 with confirmed dystrophin mutations
  • Givinostat (Duvyzat): Oral HDAC inhibitor for ambulatory patients ≥6 years

Source: Parent Project Muscular Dystrophy approved therapies overview

Common Denial Reasons & Fixes

Denial Reason How to Overturn
"Not medically necessary" Submit peer-reviewed studies showing dystrophin increase and functional benefits; include FDA approval rationale
"Experimental/investigational" Cite FDA accelerated approval status; reference CMS coverage decisions for similar cases
Missing genetic testing Provide certified lab report confirming exon 51 amenable mutation from DMD gene analysis
Non-specialist prescriber Transfer care to board-certified neurologist or obtain specialist consultation letter
Inadequate baseline assessment Complete 6MWT, NSAA, and cardiac/renal function tests at accredited DMD clinic
Insufficient corticosteroid trial Document ≥6 months therapy with pharmacy records OR medical contraindication to steroids

Appeals Process in California

California offers robust patient protections through dual regulatory oversight and binding external review.

Internal Appeal (First Level)

Timeline: File within 21 days of denial
Decision timeframe: 30 days standard, 72 hours expedited
How to file: Aetna member services or written appeal
Required: Denial letter, supporting medical records, physician statement

Independent Medical Review (IMR) - California's External Review

Eligibility: After internal appeal denial or 30-day plan non-response
Timeline: 45 days for standard IMR decision, ≤7 days for expedited
Cost: Free to patients
Authority: Department of Managed Health Care (DMHC) for HMOs; California Department of Insurance (CDI) for other plans

How to request IMR:

  1. Call DMHC Help Center: 888-466-2219
  2. Online application: healthhelp.ca.gov
  3. Submit within 6 months of final internal denial

Required documents:

  • Internal appeal denial letter
  • All medical records supporting medical necessity
  • Physician statement explaining why treatment is medically necessary
  • Any additional clinical evidence (studies, guidelines)
California Advantage: IMR decisions are binding on health plans. If approved, Aetna must authorize and pay for treatment. California patients have high success rates with rare disease IMRs.

Getting Help

DMHC Help Center: 888-466-2219 - can assist with filing complaints and IMR applications
CDI Consumer Hotline: 800-927-4357 - for plans regulated by Insurance Department
Health Consumer Alliance: Nonprofit providing free assistance with appeals

Source: California Department of Managed Health Care and Department of Insurance regulations

Switching Between DMD Treatments

Coordination Requirements

Switching from Exondys 51 to alternatives requires:

  • Genetic confirmation for other exon-skipping therapies (Amondys 45, Vyondys 53, Viltepso)
  • Specialist oversight to avoid treatment gaps
  • Insurance notification for new prior authorization if different drug class

Monitoring Considerations

  • Continue baseline assessments (6MWT, cardiac/renal function)
  • Document treatment response or lack thereof for insurance records
  • Maintain specialty pharmacy coordination through CVS Caremark or DMD-specialized providers

Counterforce Health helps patients navigate complex prior authorization requirements and appeals processes for specialty medications like DMD treatments. Their platform can assist in drafting evidence-backed appeals when initial requests are denied, particularly valuable for rare disease therapies requiring extensive documentation.

Re-trying Exondys 51 Later

If pursuing alternatives first:

  • Document treatment response to current therapy (functional assessments, side effects)
  • Maintain genetic testing records confirming exon 51 amenability
  • Track insurance changes that might affect formulary status
  • Consider resubmission if clinical status changes or new evidence emerges

Source: Based on switching protocols from Parent Project Muscular Dystrophy

Cost Support and Patient Assistance

Manufacturer Support

Sarepta Therapeutics offers:

  • Patient assistance programs for eligible uninsured/underinsured patients
  • Copay support for commercially insured patients
  • Dedicated case managers for insurance navigation

Foundation Resources

  • Parent Project Muscular Dystrophy: Advocacy and resource navigation
  • MDA (Muscular Dystrophy Association): Clinical care coordination and support services
  • CureDuchenne: Research funding and family support programs

California-Specific Programs

  • Medi-Cal coverage for eligible low-income patients
  • California Children's Services (CCS): Specialty care coordination for children with qualifying conditions
  • Covered California marketplace plans with specialty drug coverage

FAQ

Q: How long does Aetna CVS Health prior authorization take for Exondys 51 in California?
A: Standard PA decisions within 30-45 days; expedited requests ≤72 hours if prescriber documents urgent medical need.

Q: What if Exondys 51 is non-formulary on my Aetna plan?
A: Request formulary exception through prescriber with medical necessity documentation. If denied, appeal and consider California IMR.

Q: Can I request expedited appeal if my child is losing function?
A: Yes. Document functional decline and have prescriber support urgent review. California IMR expedited timeline is ≤7 days.

Q: Does step therapy apply to Exondys 51?
A: Many plans require corticosteroid trial first. Document ≥6 months therapy or medical contraindication for step therapy override.

Q: What happens if I move from another state to California?
A: California's consumer protections apply immediately. Previous denials can be appealed using California IMR process with stronger patient rights.

Q: How much does Exondys 51 cost without insurance?
A: Approximately $300,000+ annually based on weight-based dosing. Manufacturer assistance programs available for eligible patients.

Q: Can I use CVS Specialty Pharmacy for Exondys 51?
A: Yes, CVS Specialty is Aetna's preferred specialty pharmacy network. Coordinate through prescriber for prior authorization and delivery.

Q: What genetic test is required for Exondys 51?
A: DMD gene analysis confirming mutation amenable to exon 51 skipping. Must be from certified clinical genetics laboratory.

From our advocates: "We've seen families successfully appeal initial Exondys 51 denials by submitting comprehensive functional assessments alongside genetic testing. The key is demonstrating both eligibility and medical necessity through objective measures like 6-minute walk tests. California's IMR process has been particularly effective for rare disease appeals when internal appeals fail."

Counterforce Health specializes in turning insurance denials into targeted, evidence-backed appeals for complex specialty medications. Visit www.counterforcehealth.org to learn how their platform helps patients, clinicians, and specialty pharmacies get prescription drugs approved by identifying denial basis and drafting point-by-point rebuttals aligned to plan policies.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical advice. Coverage decisions vary by individual plan and circumstances. Always consult with your healthcare provider and insurance plan for personalized guidance. For assistance with California insurance appeals, contact the DMHC Help Center at 888-466-2219.

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