By Counterforce Health Team in Xenpozyme (olipudase alfa-rpcp) —

How to Get Xenpozyme (olipudase alfa-rpcp) Covered by Aetna (CVS Health) in California: Appeals Guide & Templates

Answer Box: Getting Xenpozyme Covered by Aetna (CVS Health) in California

Xenpozyme (olipudase alfa-rpcp) requires prior authorization from Aetna (CVS Health) in California. Your fastest path to approval: 1) Have your doctor submit a prior authorization request with ASMD diagnosis confirmation (enzyme assay and genetic testing), 2) Include documentation of non-CNS symptoms and failed supportive therapies, and 3) Submit via Availity provider portal or fax to 1-866-249-6155. If denied, California patients have strong appeal rights through the DMHC Independent Medical Review process, with success rates exceeding 50% for rare disease treatments.

Table of Contents

Coverage Requirements at a Glance

Requirement What It Means Where to Find It Source
Prior Authorization Required for all Xenpozyme prescriptions Aetna 2025 Precertification List Aetna
Diagnosis Confirmation ASMD confirmed by enzyme assay + genetic testing FDA label requirements FDA Approval Letter
Non-CNS Symptoms Treatment limited to non-brain manifestations Clinical policy guidelines FDA Label
Site of Care Typically requires infusion center Aetna medical policies CVS Specialty Pharmacy
Appeals Deadline 180 days from denial letter Member benefits Aetna Appeals Process

Step-by-Step: Fastest Path to Approval

1. Confirm ASMD Diagnosis

Who: Your specialist (typically hematologist or geneticist)
Document needed: Acid sphingomyelinase enzyme assay results + SMPD1 genetic testing
Timeline: 2-4 weeks for testing
Source: FDA prescribing information

2. Gather Prior Treatment Documentation

Who: Patient and care team
Document needed: Records of supportive care attempts (lipid management, organ-specific treatments)
Timeline: 1-2 days to compile
Note: Unlike other conditions, ASMD has no FDA-approved alternatives to Xenpozyme

3. Submit Prior Authorization Request

Who: Prescribing physician or staff
Document needed: California PA form
Submit via: Availity portal or fax 1-866-249-6155
Timeline: 72 hours for standard, 24 hours for urgent
Source: Aetna CA formulary

4. Include Required Clinical Information

  • ICD-10 codes: E75.244-E75.249 (acid sphingomyelinase deficiency)
  • HCPCS code: J0218 (injection, olipudase alfa-rpcp, 1 mg)
  • Baseline organ assessments (spleen/liver imaging, pulmonary function tests)
  • Weight-based dosing calculation (3 mg/kg every 2 weeks after dose escalation)

5. Monitor Decision Timeline

Standard requests: 72 hours
Expedited requests: 24 hours
Auto-approval: If Aetna doesn't respond within timeline per California regulations

Common Denial Reasons & How to Fix Them

Denial Reason How to Overturn Required Documentation
"Not medically necessary" Emphasize FDA approval for ASMD, no alternatives exist FDA approval letter, clinical trial data
"Experimental/investigational" Cite FDA approval date (August 31, 2022) FDA label, orphan drug designation
"Diagnosis not confirmed" Provide enzyme assay + genetic testing results Lab reports showing deficient ASM activity, SMPD1 variants
"Step therapy required" Document that no FDA-approved alternatives exist Medical literature search, FDA Orange Book
"Quantity limits exceeded" Justify weight-based dosing per FDA label Patient weight, dosing calculations, FDA prescribing info

Appeals Playbook for California

California offers some of the strongest patient protections in the nation for specialty drug appeals, with DMHC Independent Medical Review success rates exceeding 50% for rare disease treatments.

Internal Appeal (Level 1)

  • Timeline: File within 180 days of denial
  • Decision time: 30 days (or 72 hours for urgent)
  • Submit to: Same prior authorization fax (1-866-249-6155)
  • Required: Medical necessity letter, clinical documentation, denial letter

California Independent Medical Review (Level 2)

From our advocates: A composite case shows how thorough documentation makes the difference. One ASMD patient's initial denial was overturned at IMR when their physician included not just the genetic testing, but also serial imaging showing progressive splenomegaly and pulmonary function decline despite optimal supportive care. The independent reviewer noted that Xenpozyme was the only FDA-approved therapy for the condition.

Medical Necessity Letter Checklist

Your physician's letter should include these essential elements:

Patient Information & Diagnosis

  • Confirmed ASMD diagnosis with enzyme activity levels
  • SMPD1 genetic testing results showing pathogenic variants
  • ICD-10 codes (E75.244-E75.249)
  • Disease severity and organ involvement

Clinical Rationale

  • Non-CNS symptoms requiring treatment (hepatosplenomegaly, pulmonary dysfunction, thrombocytopenia)
  • Baseline organ function measurements
  • Treatment goals and expected outcomes
  • Monitoring plan for safety and efficacy

Evidence Base

  • FDA approval status and indication
  • Clinical trial results (ASCEND/ASCEND-Peds studies)
  • Absence of alternative FDA-approved treatments
  • Orphan drug designation

Safety & Administration

  • Dose escalation protocol to minimize infusion reactions
  • Weight-based dosing (3 mg/kg every 2 weeks)
  • Infusion center administration plan
  • Contraindication screening completed

California Independent Medical Review (IMR)

If Aetna upholds their denial, California's IMR process provides a powerful second chance. Independent physicians with expertise in rare diseases review your case and make binding decisions.

Key advantages for ASMD patients:

To request IMR:

  1. Wait for internal appeal denial or 30-day deadline
  2. Apply within 6 months at healthhelp.ca.gov
  3. Include all medical records and denial correspondence
  4. DMHC assigns to independent medical experts

Timeline: Standard IMR decisions within 45 days; expedited within 7 days for urgent cases.

Scripts & Templates

Patient Phone Script for Aetna

"Hi, I'm calling about prior authorization for Xenpozyme for my ASMD. My doctor submitted the request [date] but I haven't heard back. Can you check the status? The fax confirmation number is [X]. This is for a rare disease with only one FDA-approved treatment, so I'd like to request expedited review if possible."

Medical Necessity Letter Template Opening

"[Patient] is a [age]-year-old with genetically confirmed acid sphingomyelinase deficiency (ASMD) who requires treatment with Xenpozyme (olipudase alfa-rpcp), the only FDA-approved therapy for non-CNS manifestations of this rare lysosomal storage disorder. Enzyme assay confirmed deficient ASM activity at [level], and genetic testing revealed pathogenic variants in SMPD1 [specify variants]."

Appeal Letter Key Paragraph

"Xenpozyme received FDA approval on August 31, 2022, specifically for non-CNS manifestations of ASMD in adults and pediatric patients. Clinical trials demonstrated significant improvements in lung function and spleen size compared to placebo. No alternative FDA-approved therapies exist for this indication, making step therapy requirements inappropriate and potentially harmful due to disease progression."

Cost Assistance Programs

Sanofi Patient Assistance:

California State Resources:

For patients navigating complex insurance denials, Counterforce Health helps turn denials into targeted, evidence-backed appeals by analyzing denial letters and plan policies to draft point-by-point rebuttals aligned with payer requirements.

Frequently Asked Questions

How long does Aetna prior authorization take in California?
Standard requests: 72 hours. Expedited requests: 24 hours. If Aetna doesn't respond within these timeframes, the request is automatically approved under California regulations.

What if Xenpozyme isn't on Aetna's formulary?
You can request a formulary exception. Since no FDA-approved alternatives exist for ASMD, this strengthens your case. Include documentation that supportive care alone is inadequate.

Can I get expedited review for Xenpozyme?
Yes, if delay would seriously jeopardize your health. Progressive organ damage from untreated ASMD may qualify. Your physician must document urgency in the request.

Does step therapy apply if I haven't tried other treatments?
Step therapy typically doesn't apply since Xenpozyme is the only FDA-approved treatment for ASMD. If required, document that supportive care (current standard) has been insufficient.

What happens if my appeal is denied?
California patients can request Independent Medical Review (IMR) through DMHC. This involves independent physicians reviewing your case, with success rates exceeding 50% for rare disease treatments.

How much does Xenpozyme cost without insurance?
Wholesale acquisition cost ranges from approximately $1,515 per 4mg vial to $7,576 per 20mg vial as of January 2025. Monthly costs vary by patient weight and dosing requirements.

Can I use CVS Specialty Pharmacy for Xenpozyme?
Yes, Aetna typically requires specialty medications like Xenpozyme to be dispensed through CVS Specialty Pharmacy or other approved specialty providers.

What if I move to California from another state?
California's continuity of care laws may require Aetna to continue covering Xenpozyme if it was previously approved, provided your condition and treatment remain appropriate.

When insurance barriers seem overwhelming, remember that Counterforce Health specializes in helping patients and clinicians navigate complex specialty drug approvals by creating targeted appeals that speak directly to payer requirements and clinical evidence.

Sources & Further Reading

This guide is for informational purposes only and does not constitute medical or legal advice. Always consult with your healthcare provider and insurance plan for specific coverage decisions. For assistance with California insurance complaints, contact the DMHC Help Center at 888-466-2219.

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