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

UnitedHealthcare's Coverage Criteria for Xenpozyme in Texas: What Counts as "Medically Necessary"?

Quick Answer: Getting Xenpozyme Covered by UnitedHealthcare in Texas

UnitedHealthcare requires prior authorization for Xenpozyme (olipudase alfa-rpcp) with genetic confirmation of ASMD, specialist involvement, and baseline monitoring plan. No step therapy is required. If denied, you have 180 days for internal appeals and up to 4 months for Texas external review through an Independent Review Organization.

First step today: Contact your metabolic specialist to order genetic testing (SMPD1 mutation) or enzyme assay if not already done, then submit prior authorization through the UnitedHealthcare Provider Portal with complete documentation.

Table of Contents

  1. Policy Overview: How UnitedHealthcare Covers Xenpozyme
  2. Indication Requirements: What Diagnosis Counts
  3. Step Therapy & Exceptions: No Required Trials
  4. Quantity and Frequency Limits
  5. Required Diagnostics and Documentation
  6. Site of Care and Specialty Pharmacy Rules
  7. Evidence to Support Medical Necessity
  8. Sample "Meets Criteria" Letter Template
  9. Appeals Playbook for Texas
  10. Common Denial Reasons & How to Fix Them
  11. FAQ: Your Most Common Questions

Policy Overview: How UnitedHealthcare Covers Xenpozyme

UnitedHealthcare requires prior authorization for all Xenpozyme prescriptions across commercial, Medicare Advantage, and Medicaid plans. The drug is classified as a specialty medication due to its high cost—approximately $1,515 per 4mg vial and $7,576 per 20mg vial as of January 2025.

Plan Types and Coverage

  • Commercial HMO/PPO Plans: Standard prior authorization through OptumRx
  • Medicare Advantage: Medicare Part B coverage with prior authorization requirements
  • Medicaid: State-specific prior authorization criteria apply
  • Self-Funded Employer Plans (ERISA): Follow federal appeals process, not Texas state rules
Note: Self-funded employer plans are exempt from Texas external review laws and must use federal appeals processes instead.

All requests are processed through UnitedHealthcare's specialty pharmacy network and require electronic submission via the provider portal as of August 2025.

Indication Requirements: What Diagnosis Counts

Xenpozyme is FDA-approved for non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adults and pediatric patients. This includes Niemann-Pick disease types A and B, but excludes CNS-predominant disease.

Required Diagnostic Confirmation

UnitedHealthcare requires laboratory evidence of ASMD through:

  1. Genetic testing: Pathogenic variants in the SMPD1 gene, OR
  2. Enzyme assay: Deficient acid sphingomyelinase activity in leukocytes (gold standard)

Key Biomarkers

  • Lysosphingomyelin (LysoSM) and LysoSM 509: Elevated levels correlate with disease burden
  • Chitotriosidase: May be elevated but not required for diagnosis
  • Ceramide: Reflects impaired metabolism but not used diagnostically
Coverage Tip: Dried blood spot screening is acceptable for initial testing, but leukocyte enzyme assay provides definitive confirmation for insurance purposes.

Step Therapy & Exceptions: No Required Trials

Good news: UnitedHealthcare does not require step therapy for Xenpozyme. There are no FDA-approved alternatives for ASMD, so patients don't need to fail other treatments first.

Medical Exception Pathways

While step therapy doesn't apply, you may need to document:

  • Contraindications to supportive care measures
  • Disease progression despite optimal supportive management
  • Organ-specific complications requiring targeted enzyme replacement

Quantity and Frequency Limits

Standard Dosing Schedule

Adults (≥18 years):

  • Dose escalation over 14+ weeks: 0.1 → 0.3 → 0.6 → 1.0 → 2.0 → 3.0 mg/kg
  • Maintenance: 3 mg/kg every 2 weeks

Pediatrics (0-17 years):

  • Dose escalation over 16+ weeks: 0.03 → 0.1 → 0.3 → 0.6 → 1.0 → 2.0 → 3.0 mg/kg
  • Maintenance: 3 mg/kg every 2 weeks

UnitedHealthcare Quantity Limits

Phase Frequency Typical Authorization
Escalation Variable doses every 2 weeks 16-20 weeks initial approval
Maintenance 3 mg/kg every 2 weeks 12-month renewals
Weight adjustments Based on actual body weight (BMI ≤30) or adjusted weight (BMI >30) Requires updated documentation

Required Diagnostics and Documentation

Pre-Treatment Requirements

Laboratory Studies:

  • Liver enzymes (ALT/AST) within 1 month of initiation
  • Complete blood count with platelets
  • Comprehensive metabolic panel
  • Spleen size assessment (imaging or physical exam)
  • Pulmonary function tests (if respiratory symptoms)

Clinical Documentation:

  • Confirmed ASMD diagnosis with genetic/enzyme testing
  • Baseline organ assessments (spleen, liver, lung involvement)
  • Treatment goals and monitoring plan
  • Prescriber attestation of medical necessity

Ongoing Monitoring Requirements

  • Liver enzymes: Within 72 hours before each infusion during escalation
  • Clinical response: Regular assessment of organ size, blood counts, symptoms
  • Safety monitoring: Infusion reaction protocols and management plans
Critical: If doses are missed, re-escalation may be required before resuming maintenance therapy.

Site of Care and Specialty Pharmacy Rules

Approved Administration Sites

  1. Specialty infusion centers (preferred)
  2. Clinic/office settings with appropriate monitoring
  3. Home infusion (maintenance phase only, with healthcare supervision)
  4. Outpatient hospital (requires medical necessity documentation)

Specialty Pharmacy Requirements

  • Must use UnitedHealthcare/OptumRx in-network specialty pharmacy
  • Out-of-network providers typically result in coverage denials
  • Prior authorization must specify both drug supply and administration site

Home Infusion Criteria

  • Patient must be on stable maintenance dose (3 mg/kg)
  • Demonstrated tolerance to infusions
  • Qualified healthcare provider supervision
  • Emergency management plan in place

Evidence to Support Medical Necessity

Primary Evidence Sources

  1. FDA labeling: Xenpozyme prescribing information
  2. Manufacturer guidelines: Dosing and administration protocols
  3. Peer-reviewed literature: Studies demonstrating efficacy in ASMD
  4. Specialty society recommendations: Lysosomal storage disease treatment guidelines

Key Clinical Evidence Points

  • Organ involvement: Document spleen size, liver function, pulmonary symptoms
  • Disease progression: Show worsening without treatment
  • Quality of life impact: Functional limitations, exercise intolerance
  • Specialist consultation: Metabolic disease or genetics specialist involvement

Sample "Meets Criteria" Letter Template

Patient [Name], DOB [Date], has confirmed acid sphingomyelinase deficiency 
(ASMD) based on [genetic testing showing SMPD1 mutations / enzyme assay 
demonstrating deficient ASM activity]. Clinical manifestations include 
[hepatosplenomegaly/thrombocytopenia/pulmonary infiltrates] consistent 
with non-CNS ASMD.

Baseline assessments show [spleen size, liver enzymes, platelet count]. 
The patient has been evaluated by [metabolic specialist name] and meets 
FDA criteria for Xenpozyme therapy. Treatment will follow the recommended 
dose escalation protocol with regular monitoring of liver enzymes and 
clinical response.

No contraindications to therapy exist. The patient/family understands 
the infusion schedule and monitoring requirements. Request approval for 
Xenpozyme 3 mg/kg every 2 weeks following dose escalation, dispensed 
through [specialty pharmacy] and administered at [infusion center].

Appeals Playbook for Texas

If UnitedHealthcare denies your Xenpozyme request, Texas law provides strong appeal rights with specific timelines and processes.

Internal Appeals Process

Step Timeline Requirements Submission Method
File appeal 180 days from denial Denial letter, clinical evidence, medical necessity letter Electronic submission required
Standard review 30 days for decision Complete documentation Provider Portal
Expedited review 72 hours for urgent cases Medical urgency documentation Same portal, mark urgent

Texas External Review (IRO)

After exhausting internal appeals:

Filing Requirements:

Decision Timeline:

  • Standard cases: 20 days from IRO assignment
  • Urgent cases: 5 days for life-threatening situations
  • Emergency cases: 3 days for current treatments being denied

IRO Decision: Binding on UnitedHealthcare for state-regulated plans

Texas Help Resources:Texas Department of Insurance: 1-800-252-3439Office of Public Insurance Counsel: 1-877-611-6742IRO Information Line: 1-866-554-4926

Common Denial Reasons & How to Fix Them

Denial Reason Fix Strategy Required Documentation
No ASMD diagnosis Submit genetic testing or enzyme assay SMPD1 gene results or ASM activity levels
CNS disease indication Clarify non-CNS manifestations Specialist letter emphasizing systemic disease
Missing specialist consultation Get metabolic disease specialist evaluation Consultation note and treatment recommendation
Inadequate monitoring plan Submit detailed safety protocol Liver enzyme monitoring schedule, infusion protocols
Site of care issues Document medical necessity for specific site Justification for home vs. center administration
Quantity over limits Provide weight-based dosing calculation Current weight, dosing rationale, escalation plan

When to Get Help from Counterforce Health

Navigating UnitedHealthcare's prior authorization process for specialty medications like Xenpozyme can be complex and time-consuming. Counterforce Health specializes in turning insurance denials into successful appeals by analyzing denial letters, identifying specific coverage criteria, and crafting evidence-based rebuttals that speak directly to each payer's requirements.

If you're facing a Xenpozyme denial or want to strengthen your initial prior authorization, Counterforce Health can help ensure your submission includes all the clinical evidence, proper documentation, and payer-specific language needed for approval. Their platform is designed specifically for rare disease medications and complex specialty drug appeals.

FAQ: Your Most Common Questions

Q: How long does UnitedHealthcare prior authorization take for Xenpozyme in Texas? A: Standard decisions are issued within 72 hours of complete submission. Expedited reviews for urgent cases are processed within 24-72 hours.

Q: What if Xenpozyme isn't on my formulary? A: Xenpozyme requires prior authorization regardless of formulary status. Non-formulary placement doesn't prevent coverage if medical necessity is established.

Q: Can I request an expedited appeal in Texas? A: Yes, both internal appeals and external IRO reviews can be expedited if delay would jeopardize your health. Document medical urgency clearly.

Q: Does step therapy apply if I've tried treatments outside Texas? A: UnitedHealthcare doesn't require step therapy for Xenpozyme since no alternatives exist. Previous treatment history may support medical necessity but isn't required.

Q: What happens if I miss doses during escalation? A: Missing doses may require restarting escalation protocol. Work with your provider to maintain the schedule and notify UnitedHealthcare of any changes.

Q: Can I switch from hospital outpatient to home infusion? A: Yes, but requires prior authorization update documenting safety criteria for home administration during maintenance phase.

Q: How often do I need reauthorization? A: Typically every 12 months, with documentation of clinical benefit, adherence to monitoring, and absence of serious adverse events.

Q: What if my employer plan is self-funded? A: Self-funded ERISA plans follow federal appeals rules, not Texas external review. Check your plan documents or contact HR to confirm plan type.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Coverage policies and appeal procedures may vary by plan and change over time. Always consult your healthcare provider for medical decisions and verify current procedures with UnitedHealthcare and Texas regulatory agencies. For personalized assistance with complex appeals, consider consulting with Counterforce Health or other qualified advocacy services.

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