By Counterforce Health Team in Kanuma (sebelipase alfa) —

UnitedHealthcare's Coverage Criteria for Kanuma (Sebelipase Alfa) in Ohio: What Counts as "Medically Necessary"?

Answer Box: Getting Kanuma Covered by UnitedHealthcare in Ohio

UnitedHealthcare covers Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency (LAL-D) with prior authorization required. To qualify, you need: confirmed LAL-D diagnosis (enzyme deficiency or LIPA gene variants), documented hepatic abnormalities (elevated ALT/AST, hepatomegaly), and evidence of failed supportive therapy (diet, statins). First step: Have your specialist submit PA via UHC Provider Portal with complete diagnostic records and failure documentation. Appeals available through Ohio's external review process if denied.

Table of Contents

Policy Overview: How UnitedHealthcare Covers Kanuma

UnitedHealthcare Community Plan of Ohio classifies Kanuma as a medical benefit requiring prior authorization for all plan types (HMO, PPO, and Medicaid). The drug is covered under their Medical Policy for Enzyme Deficiency Therapies, effective through 2025.

Coverage Status at a Glance

Requirement Status Documentation Needed
Prior Authorization Required PA form + clinical records
Formulary Status Covered with PA No step therapy required
Age Restrictions ≥1 month of age Birth certificate/medical records
Prescriber Requirements Specialist only Hepatologist, geneticist, or metabolic specialist
Site of Care Specialty pharmacy + infusion center Contracted network only

Where to Find Official Policies: UnitedHealthcare provider portal under "Medical Policies" or the Ohio-specific enzyme deficiency policy.

Indication Requirements: Proving LAL-D Diagnosis

UnitedHealthcare requires definitive LAL-D diagnosis through objective testing—clinical symptoms alone won't qualify.

Diagnostic Confirmation (Must Have One)

Option 1: Enzyme Testing

  • LAL enzyme activity assay showing deficiency
  • Acceptable methods: dried blood spot (DBS) or whole blood with Lalistat-2 inhibitor
  • Typical cutoff: <1.5 nmol/h/mL (verify with lab's reference range)
  • Must be from certified laboratory

Option 2: Genetic Testing

  • Biallelic pathogenic variants in LIPA gene
  • Full gene sequencing with deletion/duplication analysis
  • Report must specify "pathogenic" or "likely pathogenic" variants
  • Carrier status (one variant) doesn't qualify

Clinical Evidence Requirements

Patient must demonstrate at least one LAL-D manifestation:

  • Dyslipidemia (elevated LDL-C, triglycerides, low HDL-C)
  • Elevated liver enzymes (ALT, AST above normal range)
  • Hepatomegaly or splenomegaly on imaging/exam
  • Growth failure or malabsorption (pediatric patients)
  • Adrenal calcification (Wolman disease)
Tip: Order both enzyme and genetic testing when possible. Having both strengthens your case and provides backup if one result is questioned.

Step Therapy & Medical Exceptions

Good news: Kanuma is exempt from step therapy due to its orphan drug status for ultra-rare LAL-D. However, UnitedHealthcare requires documentation that standard supportive therapies have been tried and failed.

Required "Failure" Documentation

Lipid Management Attempts:

  • Low-fat, low-cholesterol diet (document dietitian involvement, duration)
  • Statin therapy with specific agent, dose, duration, and lipid response
  • Other lipid-lowering agents if tried (ezetimibe, fibrates, etc.)

Evidence of Inadequate Response:

  • Persistent elevated LDL-C despite maximum tolerated statin
  • Continued liver enzyme elevation
  • Progressive hepatomegaly or liver dysfunction
  • Intolerance to conventional therapy (document side effects)

Medical Exception Pathway

If you haven't tried conventional therapy due to contraindications:

  • Document specific contraindications (e.g., statin intolerance, severe liver disease)
  • Provide clinical rationale why delay would be harmful
  • Include specialist attestation of urgency

Quantity Limits & Dosing Rules

UnitedHealthcare enforces weight-based quantity limits aligned with FDA labeling.

Approved Dosing Regimens

Rapidly Progressive Disease (infants <6 months):

  • Initial: 1 mg/kg IV weekly
  • May increase to 3 mg/kg weekly if suboptimal response
  • Maximum: 5 mg/kg weekly with strong justification

All Other Patients (pediatric and adult):

  • Standard: 1 mg/kg IV every other week
  • May increase to 3 mg/kg every other week if needed

Quantity Calculation

  • Each vial contains 20 mg/10 mL
  • Round up to next whole vial for patient's weight
  • Example: 70 kg patient at 1 mg/kg = 70 mg = 4 vials per infusion

Reauthorization: Required every 6-12 months with evidence of clinical response (improved liver enzymes, lipids, or growth parameters).

Required Diagnostics & Documentation

Essential Lab Work

Baseline Requirements:

  • Complete lipid panel (LDL-C, HDL-C, triglycerides, total cholesterol)
  • Liver function tests (ALT, AST, bilirubin, alkaline phosphatase)
  • LAL enzyme activity or genetic testing results
  • Growth charts (pediatric patients)

Imaging Studies:

  • Abdominal ultrasound or CT showing hepatomegaly
  • Liver MRI if available (shows characteristic lipid accumulation)

Timing Requirements:

  • Diagnostic tests: within 6 months of PA submission
  • Supporting labs: within 3 months
  • Clinical notes: current visit documenting ongoing symptoms

Documentation Quality Tips

For Enzyme Results:

  • Include numeric value, reference range, and lab name
  • Note collection method (DBS vs. whole blood)
  • Attach full lab report, not just summary

For Genetic Testing:

  • Provide complete report with variant classification
  • Include inheritance pattern and clinical significance
  • Document family history if relevant

Site of Care & Specialty Pharmacy Requirements

Specialty Pharmacy Network

Kanuma must be dispensed through UnitedHealthcare's contracted specialty pharmacy network:

  • Optum Specialty Pharmacy is typically the preferred provider
  • Cold-chain shipping required due to refrigeration needs
  • Coordinate with infusion center for delivery timing

Approved Infusion Sites

Acceptable Locations:

  • Hospital outpatient infusion centers
  • Contracted ambulatory infusion centers
  • Certified home infusion services (with emergency protocols)

Requirements:

  • Must have emergency equipment for anaphylaxis management
  • Staff trained in enzyme replacement therapy infusions
  • Access to emergency medical services
Note: Some plans require "white bagging" (specialty pharmacy ships directly to infusion site) rather than "brown bagging" (patient picks up drug). Verify your plan's policy.

Evidence to Support Medical Necessity

Key Clinical Guidelines

FDA Prescribing Information:

  • Kanuma approved for LAL-D in patients ≥1 month
  • Demonstrated efficacy in reducing liver volume and improving lipids
  • Safety profile established in clinical trials

Peer-Reviewed Literature:

  • ARISE trial (New England Journal of Medicine): showed significant improvements in liver enzymes and lipid profiles
  • Long-term extension studies documenting sustained benefits
  • Pediatric data supporting early intervention

How to Cite Effectively

In Your PA Letter:

  • "Per FDA labeling, sebelipase alfa is indicated for LAL deficiency treatment"
  • "Clinical trials demonstrate 30-40% reduction in liver volume"
  • "No alternative enzyme replacement therapies exist for LAL-D"

Attach Key References:

  • FDA prescribing information
  • Primary efficacy study abstracts
  • Relevant clinical practice guidelines

Sample "Meets Criteria" Narrative

"This [age]-year-old patient has confirmed lysosomal acid lipase deficiency based on [enzyme activity of X nmol/h/mL, reference range Y] and/or [biallelic pathogenic LIPA variants]. Clinical manifestations include persistent dyslipidemia (LDL-C [value] mg/dL), elevated liver enzymes (ALT [value], AST [value]), and hepatomegaly on imaging. Despite optimized supportive therapy including [specific diet/medications tried, duration, response], the patient continues to have [specific ongoing abnormalities]. Enzyme replacement therapy with sebelipase alfa is medically necessary to address the underlying enzymatic deficiency, prevent progression of liver disease, and reduce cardiovascular risk. The requested dosing of [X mg/kg every other week/weekly] aligns with FDA labeling for LAL-D treatment."

Common Denial Reasons & How to Fix Them

Denial Reason How to Overturn Documents Needed
"Diagnosis not confirmed" Submit objective test results LAL enzyme assay + genetic testing reports
"Insufficient prior therapy" Document specific failures Treatment timeline with doses, duration, response
"Not medically necessary" Emphasize disease progression Serial labs showing worsening, imaging, specialist notes
"Experimental/investigational" Cite FDA approval FDA label, clinical trial data, guidelines
"Incorrect dosing" Verify weight-based calculation Current weight, dosing calculation, FDA reference

Quick Fixes for Common Issues

Missing Specialist Requirement:

  • Obtain referral to hepatologist, geneticist, or metabolic specialist
  • Have specialist co-sign PA request and provide supporting letter

Incomplete Diagnostic Workup:

  • Order missing tests (enzyme activity if only genetic testing done)
  • Ensure all results are within required timeframes

Inadequate Prior Therapy Documentation:

  • Create timeline showing specific agents, doses, duration, and outcomes
  • Include pharmacy records or prescription history if available

Appeals Process for Ohio Residents

If your initial PA is denied, Ohio residents have robust appeal rights through both UnitedHealthcare's internal process and the state's external review system.

Internal Appeals (UnitedHealthcare)

First-Level Appeal:

  • Deadline: 21 calendar days from denial notice
  • Submission: UHC Provider Portal or OptumRx system
  • Timeline: Decision within 5-10 business days (72 hours if urgent)

Second-Level Appeal:

  • Available if first appeal denied
  • Same submission process and timelines
  • Includes independent medical review

External Review (Ohio Department of Insurance)

When to Use:

  • After exhausting internal appeals
  • For medical necessity or experimental/investigational denials
  • When UHC misses internal appeal deadlines

Process:

  • Deadline: 180 days from final internal denial
  • Contact: Ohio Department of Insurance at 1-800-686-1526
  • Timeline: 30 days for standard review, 72 hours for urgent cases
  • Authority: Decision is binding on UnitedHealthcare

Required Documentation:

  • All previous appeal materials
  • Updated clinical information
  • Specialist letter emphasizing medical necessity
  • Peer-reviewed literature supporting treatment
Important: Even if UnitedHealthcare claims your case isn't eligible for external review, Ohio's Department of Insurance can independently determine eligibility and order a review.

Getting Help with Appeals

Ohio Resources:

For complex cases requiring comprehensive appeal support, Counterforce Health specializes in turning insurance denials into successful appeals by analyzing denial letters, plan policies, and clinical notes to create targeted, evidence-backed rebuttals that align with payer-specific requirements.

FAQ: Your Top Questions Answered

How long does UnitedHealthcare PA take in Ohio? Standard PA decisions: 5-10 business days. Urgent/expedited requests (when delay would jeopardize health): 72 hours. Submit as urgent if patient has rapidly progressive disease or liver failure risk.

What if Kanuma is non-formulary on my plan? Kanuma is covered with PA on UnitedHealthcare Community Plan of Ohio. For other UHC plans, request a formulary exception citing lack of alternatives and FDA approval for LAL-D.

Can I request an expedited appeal? Yes, if your physician certifies that delay would seriously jeopardize life, health, or ability to regain maximum function. This applies to both internal appeals and external review.

Does step therapy apply if I failed treatments outside Ohio? Prior therapy from any location counts. Provide documentation of previous treatment attempts, responses, and reasons for failure or intolerance.

What if I can't afford Kanuma even with insurance? Contact Alexion's patient assistance program and explore rare disease foundation grants. Some programs provide free drug for eligible patients regardless of insurance status.

How often do I need reauthorization? Typically every 6-12 months. Include updated labs showing clinical response (improved liver enzymes, lipids, growth parameters) and current weight for dosing verification.

What happens if UnitedHealthcare changes their policy? Existing patients usually have "grandfathering" protection. Monitor policy updates and work with your specialist to ensure continued coverage under any new criteria.

Can I switch to a different infusion center? Yes, but the new center must be in UnitedHealthcare's network and meet their requirements for enzyme replacement therapy administration. Coordinate with Optum Specialty Pharmacy for delivery logistics.

Medical Disclaimer: This information is for educational purposes only and doesn't constitute medical advice. Always consult your healthcare provider for personalized medical guidance. Insurance policies change frequently—verify current requirements with UnitedHealthcare and the Ohio Department of Insurance.

Need Help with Your Appeal? Counterforce Health helps patients, clinicians, and specialty pharmacies get prescription drugs approved by creating targeted appeals that address specific denial reasons with evidence-backed rebuttals aligned to each plan's requirements.

Sources & Further Reading

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