By Counterforce Health Team in Translarna (ataluren) —

How to Get Translarna (Ataluren) Covered by Cigna in California: Complete Prior Authorization Guide

Answer Box: Getting Translarna (Ataluren) Covered by Cigna in California

Translarna (ataluren) is not FDA-approved but may be available through expanded access programs. For Cigna coverage in California: (1) Submit prior authorization via Cigna's California-specific form to fax 1-866-873-8279, (2) Include genetic testing confirming nonsense mutation DMD and ambulatory status documentation, (3) If denied, file internal appeal within 180 days, then request California Independent Medical Review (IMR) for external review. Success rates for specialty drug appeals are approximately 10% in California IMR.

Table of Contents

  1. Policy Overview: Cigna Coverage in California
  2. Medical Necessity Requirements
  3. Step Therapy and Exceptions
  4. Quantity and Frequency Limits
  5. Required Diagnostics and Documentation
  6. Specialty Pharmacy Requirements
  7. Evidence to Support Medical Necessity
  8. Appeals Process in California
  9. Common Denial Reasons and Solutions
  10. Costs and Patient Support
  11. FAQ

Policy Overview: Cigna Coverage in California

Cigna operates multiple plan types in California, each with different coverage rules for specialty medications like Translarna (ataluren). Understanding your specific plan type is crucial for navigating the authorization process.

Plan Types and Coverage Pathways

Commercial Plans (HMO/PPO): Most Cigna commercial plans in California require specialty medications to be filled through Accredo specialty pharmacy. Prior authorization is typically required for all specialty drugs not on the formulary.

Medicare Advantage: Cigna Medicare plans follow CMS guidelines with additional plan-specific requirements. These plans have higher appeal rates (approximately 18%) for prior authorization denials.

Medicaid (Medi-Cal): Coverage varies based on whether Cigna is your managed care plan through Medi-Cal.

Note: Translarna is not listed on Cigna's 2025 National Preferred Formulary, making formulary exception requests necessary.

Medical Necessity Requirements

Since Translarna remains under FDA review following a resubmitted New Drug Application (NDA), Cigna's medical necessity criteria focus on clinical justification and regulatory status.

Core Requirements

Requirement Documentation Needed Source
Genetic confirmation Lab report showing nonsense mutation in DMD gene DMD genetic testing protocols
Ambulatory status Clinical assessment confirming patient can walk Clinical notes from neuromuscular specialist
Age criteria Patient ≥2 years old Based on historical EU approval criteria
Specialist prescriber Neurologist or neuromuscular specialist Cigna PA requirements

Genetic Testing Documentation

Genetic testing must demonstrate a nonsense mutation causing Duchenne muscular dystrophy. According to established protocols, testing should include:

  • Initial screening for large deletions/duplications (accounts for ~65% of DMD cases)
  • Sequencing analysis if no large mutations found (covers remaining 25-30% including nonsense mutations)
  • Exact mutation position documentation for correlation with clinical phenotype

Nonsense mutations account for approximately 15% of dystrophinopathy patients and require specific documentation as they determine eligibility for mutation-specific therapies.

Step Therapy and Exceptions

Currently, there are no FDA-approved treatments specifically for nonsense mutation DMD, which affects step therapy requirements.

Standard DMD Treatments

Before considering Translarna, insurers typically expect documentation of:

  1. Corticosteroids (prednisone, deflazacort) - first-line therapy
  2. Supportive care including physical therapy, cardiac monitoring
  3. Other approved DMD therapies if genetically appropriate (exon-skipping drugs for eligible mutations)

Medical Exception Pathways

If standard treatments are contraindicated or ineffective, document:

  • Contraindications to corticosteroids (growth concerns, behavioral issues, bone health)
  • Treatment failures with specific outcomes and duration
  • Clinical deterioration despite standard care

Quantity and Frequency Limits

Translarna dosing in clinical studies typically follows:

  • Morning and midday doses: 10 mg/kg each
  • Evening dose: 5 mg/kg
  • Total daily dose: 25 mg/kg divided into three doses
  • Supply limits: Typically 30-day supplies for specialty medications

Coverage requests should specify exact dosing based on patient weight and include rationale for dose selection.

Required Diagnostics and Documentation

Essential Clinical Documentation

Genetic Testing Results:

  • Laboratory report confirming nonsense mutation
  • Mutation position and predicted protein effect
  • Laboratory certification and methodology used

Clinical Assessment:

  • Neuromuscular examination documenting ambulatory status
  • Functional assessments (6-minute walk test, North Star Ambulatory Assessment)
  • Cardiac and pulmonary function evaluations

Prior Treatment History:

  • Corticosteroid trials with outcomes
  • Physical therapy records
  • Other DMD-specific interventions attempted
Tip: Ensure genetic testing uses MLPA-based methods combined with sequencing for highest mutation detection rates (~99%).

Specialty Pharmacy Requirements

Cigna requires most specialty medications to be dispensed through Accredo specialty pharmacy.

Accredo Process

  1. Prescription submission by prescriber to Accredo
  2. Prior authorization handled by Accredo on behalf of patient
  3. Delivery coordination directly to patient or clinic
  4. Ongoing monitoring and refill management

Contact Information:

  • Phone: 877-826-7657
  • Hours: Monday-Friday 7am-10pm CST, Saturday 7am-4pm CST

Evidence to Support Medical Necessity

Since Translarna lacks FDA approval, supporting evidence becomes critical for coverage decisions.

Key Clinical Evidence

Published Studies:

  • STRIDE registry results showing disease progression benefits
  • Phase 3 Study 041 outcomes
  • Real-world evidence from international use

Regulatory Support:

  • FDA acceptance of resubmitted NDA in late 2024
  • Historical conditional approval in EU (though subsequently withdrawn)
  • Orphan drug designation status

Clinical Guidelines:

  • International consensus statements on DMD care
  • Neuromuscular specialist recommendations
  • Patient advocacy organization position statements

Appeals Process in California

California offers robust appeal rights through both internal insurer processes and external review.

Internal Appeals with Cigna

Timeline: 180-day window to file internal appeal after denial Process: Submit written appeal with additional clinical documentation Expedited review: Available for urgent medical situations

California Independent Medical Review (IMR)

After internal appeal denial, California residents can request external review through the Department of Managed Health Care.

IMR Process:

  1. File grievance with Cigna first (30-day response required)
  2. Request IMR if grievance denied or no response
  3. Independent physician review by specialists in relevant field
  4. Binding decision within 45 days (7 days for expedited)
Important: IMR success rates for specialty drugs are approximately 10% based on recent California data, but cases with strong clinical evidence may have better outcomes.

When dealing with Translarna appeals, Counterforce Health helps patients and clinicians turn insurance denials into targeted, evidence-backed appeals. The platform ingests denial letters and clinical notes, then identifies the specific denial basis and drafts point-by-point rebuttals aligned to the plan's own rules, pulling the right citations and clinical facts to strengthen your case.

Common Denial Reasons and Solutions

Denial Reason Solution Strategy Required Documentation
Not FDA-approved Emphasize NDA under review, clinical evidence FDA correspondence, clinical studies
Experimental/investigational Cite international approvals, published data EMA documents, peer-reviewed studies
Lack of medical necessity Strengthen clinical justification Specialist letters, functional assessments
Step therapy not met Document contraindications to alternatives Treatment history, adverse events

Costs and Patient Support

Manufacturer Support Programs

PTC Therapeutics may offer patient assistance programs. Contact the manufacturer directly for current program availability and eligibility requirements.

Foundation Support

  • Patient Advocate Foundation: Provides copay assistance and case management
  • HealthWell Foundation: Offers grants for specialty medication copays
  • CDF's Compass Program: Assists with insurance navigation

California-Specific Resources

  • Health Consumer Alliance: Free insurance assistance for California residents
  • California Department of Insurance: Consumer hotline at 800-927-4357

FAQ

How long does Cigna prior authorization take in California? Standard prior authorization decisions are made within 72 hours for non-urgent requests, 24 hours for urgent requests, per California regulations.

What if Translarna is not on Cigna's formulary? You can request a formulary exception through your prescriber, providing clinical justification for why formulary alternatives are inappropriate.

Can I request an expedited appeal if my condition is worsening? Yes, both Cigna internal appeals and California IMR offer expedited review processes for urgent medical situations.

Does Translarna require specialty pharmacy dispensing? Yes, Cigna typically requires specialty medications like Translarna to be filled through Accredo specialty pharmacy for coverage.

What genetic testing is required for coverage? Documentation must confirm a nonsense mutation in the DMD gene causing Duchenne muscular dystrophy, with specific mutation position identified.

How much does Translarna cost without insurance? International pricing suggests very high annual costs (six-figure range), though exact U.S. pricing is not yet established pending FDA approval.

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 plan directly for coverage determinations. For assistance with complex appeals, Counterforce Health provides specialized support in turning insurance denials into successful, evidence-backed appeals.

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