Kanuma (sebelipase alfa) — HCPCS J2840

First-in-class FDA approval — LAL-D has no class peer FDA verified May 2026

Kanuma (FDA-approved December 8, 2015) is the only enzyme replacement therapy ever approved for lysosomal acid lipase deficiency.

Lysosomal acid lipase deficiency (LAL-D) is a rare autosomal recessive lysosomal storage disorder caused by biallelic pathogenic variants in LIPA. The deficient enzyme normally hydrolyzes cholesteryl esters and triglycerides delivered to the lysosome; without it, substrate accumulates in liver, spleen, intestine, lymph nodes, and vascular walls. Clinical presentation falls on a phenotypic spectrum from rapidly progressive infantile disease (Wolman) — uniformly fatal in the first year of life without ERT — to late-onset disease (cholesteryl ester storage disease, CESD) presenting from childhood to adulthood with dyslipidemia, hepatomegaly, hepatic fibrosis, and accelerated atherosclerotic cardiovascular disease.

Lysosomal storage disorder (LSD) ERT landscape

Kanuma joins a small set of recombinant ERTs that each target one specific LSD enzyme deficiency. These products are not interchangeable — each has a unique HCPCS, NDC, and clinical indication.

Pivotal trial — ARISE (CESD)

The ARISE trial (Acid Lipase Replacement Investigating Safety and Efficacy) was the pivotal Phase 3 randomized double-blind placebo-controlled study in 66 children and adults with CESD. After 20 weeks of therapy at 1 mg/kg q2w, sebelipase alfa significantly improved ALT normalization, LDL-C, non-HDL-C, HDL-C, triglycerides, and hepatic fat content versus placebo. The Wolman indication was supported by an open-label single-arm trial in infants with rapidly progressive LAL-D, demonstrating substantial survival benefit versus the natural-history comparator (uniformly fatal disease in the first year).

Dosing & unit math FDA label verified Dec 2015 rev

From FDA prescribing information for Kanuma (sebelipase alfa).

Wolman disease — rapidly progressive infantile LAL-D

• Starting dose: 1 mg/kg IV weekly
• First escalation: Increase to 3 mg/kg weekly if suboptimal clinical response (growth, weight gain, transaminase trends, ferritin, ALT/AST normalization) per prescriber judgment
• Second escalation: Further increase to 5 mg/kg weekly if response remains suboptimal
• Infants typically initiate therapy in NICU or PICU under intensive monitoring; severe disease and infusion reaction risk require emergency response capability
• Lifelong therapy

Cholesteryl ester storage disease (CESD) — late-onset LAL-D

• Dose: 1 mg/kg IV every 2 weeks
• Same dose for children, adolescents, and adults — weight-based throughout
• Per ARISE trial: 1 mg/kg q2w produces ALT normalization, LDL-C reduction, and hepatic fat reduction
• Lifelong therapy

Unit math — the 1 mg billing unit

1 J2840 billing unit = 1 mg of sebelipase alfa. Total mg administered equals the unit count entered on the claim. Only one vial size exists (20 mg/10 mL), so weight-based dosing virtually always produces partial-vial waste — report on a separate JW line.

Worked example — CESD adult (70 kg patient, 1 mg/kg q2w)

Worked example — Wolman infant (5 kg infant, 1 mg/kg weekly → escalation to 3 mg/kg)

NDC reference FDA NDC Directory verified May 2026

Administration codes CPT verified May 2026

Kanuma is non-chemo enzyme replacement therapy — therapeutic IV codes only.

Modifiers CMS verified May 2026

JZ + JW — both apply on virtually every claim

Kanuma is uniquely vulnerable to fixed-vial waste: only one vial size (20 mg) exists, but dosing is weight-based across infants, children, and adults at multiple mg/kg levels. A 70 kg adult at 1 mg/kg (CESD) needs 70 mg but must draw 4 vials (80 mg), leaving 10 mg waste; a 5 kg infant at 1 mg/kg (Wolman starting dose) needs only 5 mg but must draw one full 20 mg vial, leaving 15 mg waste. Bill JW with the discarded mg on a separate claim line, AND JZ on the administered mg per CMS's July 2023 single-dose container policy.

Modifier 25 — same-day E/M

Use modifier 25 on the E/M code when a significant, separately identifiable evaluation and management service is performed on the same day as the infusion. Routine pre-infusion clinical assessment is bundled into the infusion service. Wolman infants frequently have same-day specialist E/M documentation supporting modifier 25.

340B modifiers (JG, TB)

For 340B-acquired Kanuma, follow your MAC's current 340B modifier policy. Alexion / AZ Access 360 does not provide 340B-specific billing instructions; defer to MAC LCD and your hospital's 340B compliance team.

ICD-10-CM diagnosis coding FY2026 verified May 2026

E75.5 is the primary anchor; supplementary codes document organ-system findings supporting medical necessity.

Site of care & place of service Verified May 2026

Site-of-care utilization management on Kanuma differs sharply by phenotype. Wolman infants are managed in NICU/PICU or pediatric hospital outpatient settings throughout early therapy. Stable CESD adults are routinely steered out of HOPD into office, ambulatory infusion suite, or home infusion settings after a documented track record of incident-free infusions.

Claim form field mapping Alexion / AZ Access 360 2025

From Alexion Kanuma reimbursement guide (AZ Access 360 HCP materials, post-AstraZeneca integration).

Payer policy snapshot Reviewed May 2026

All major payers gate Kanuma behind diagnostic confirmation, specialist consultation, and phenotype-matched dosing. As the only LAL-D treatment, formulary brand selection is not in play.

Step therapy

Step therapy is not applicable — there is no alternative LAL-D therapy to step through. Statin therapy is supportive only in CESD and is not a payer-acceptable step before Kanuma in symptomatic patients meeting label criteria. Some payers may require documentation of statin trial or intolerance, but this is variable and clinically secondary.

Reauthorization

Annual reauthorization is the norm. For CESD, submit current ALT/AST, LDL-C, HDL-C, triglycerides, hepatic imaging (US, MRI elastography, or FibroScan) showing stability or improvement. For Wolman, submit weight gain / growth percentiles, transaminase trends, ferritin, hepatosplenic volume, and clinical narrative. Documented stability or improvement on therapy supports continued coverage. Document any dose escalations (Wolman 1 → 3 → 5 mg/kg) with the clinical rationale.

Medicare reimbursement CMS Q2 2026 (live)

Quarterly ASP from CMS Part B Drug Pricing File. Refreshes automatically each quarter.

Coverage

Kanuma is covered by all MACs under their generic Part B drug-coverage framework with appropriate E75.5 + diagnostic documentation. As of May 2026, no NCD specific to sebelipase alfa; local MAC LCDs may exist for rare-disease ERTs. Verify per jurisdiction. Wolman infants are most often Medicaid-covered rather than Medicare-covered — coordinate state Medicaid PA workflow with the family early.

Code history

• J2840 — permanent code, descriptor "Injection, sebelipase alfa, 1 mg"; assigned after FDA approval (December 8, 2015)

Next ASP update

ASP is updated quarterly by CMS. Next update: July 1, 2026 for Q3 2026 pricing (effective July 1 – September 30, 2026).

Patient assistance — Alexion OneSource & AZ Access 360 Alexion / AZ verified May 2026

• Alexion OneSource (now operating under AstraZeneca Access 360 post-2021 acquisition): 1-888-765-4747 — benefits investigation, prior authorization assistance, appeal support, copay program enrollment, free-drug program for eligible patients, specialty pharmacy coordination
• Kanuma Patient Support: dedicated case management for LAL-D patients; coordinates NICU/PICU logistics for Wolman infants, ambulatory infusion suite setup for CESD adults, and home infusion transitions
• Commercial copay assistance: available for eligible commercially-insured patients (excludes Medicare, Medicaid, federal program patients per federal anti-kickback rules)
• Patient assistance program (PAP): free product for uninsured / underinsured patients meeting income criteria
• Foundations (Medicare / Medicaid patients): PAN Foundation (verify open lysosomal storage disease funds quarterly), HealthWell Foundation, National Organization for Rare Disorders (NORD) patient assistance programs
• Web: azaccess360.com · kanuma.com (patient-facing)

Common denials & how to fix them

Frequently asked questions

What is the HCPCS code for Kanuma?

Kanuma (sebelipase alfa) is billed under HCPCS J2840 — "Injection, sebelipase alfa, 1 mg." One billing unit equals 1 mg. A 1 mg/kg dose for a 70 kg adult with CESD = 70 mg = 70 units. J2840 is a permanent code added after FDA approval in December 2015.

Is Kanuma dosing different for Wolman disease versus CESD?

Yes — phenotype determines dose and frequency. CESD (late-onset LAL-D) is dosed 1 mg/kg IV every 2 weeks. Rapidly progressive Wolman disease (infantile LAL-D) starts at 1 mg/kg IV weekly and may be escalated to 3 mg/kg weekly and then to 5 mg/kg weekly based on clinical response. The phenotype must be documented on the PA — payers will deny a Wolman-frequency claim under a CESD diagnosis or vice versa.

Is LAL enzyme assay required for Kanuma prior authorization?

Yes — confirmed lysosomal acid lipase (LAL) enzyme deficiency on a validated assay (typically dried blood spot for screening, confirmed in lymphocytes or fibroblasts) is the universal PA gate. Most payers also require LIPA gene mutation analysis. Submitting a Kanuma PA without the LAL enzyme assay result is the #1 cause of initial denial. Order the enzyme assay AND genotype before submitting the PA, not after.

Is LIPA genotype required for Kanuma?

Most major payers require both LAL enzyme assay AND LIPA gene mutation analysis (biallelic pathogenic variants) for diagnostic confirmation. Some payers will accept the enzyme assay alone with strong specialist documentation, but biallelic LIPA variants are the molecular gold standard and resolve the most ambiguous cases. Order both at the same reference lab when possible.

What administration CPT do I use for Kanuma?

96365 (initial therapeutic IV infusion, up to 1 hour) plus 96366 (each additional hour) for the standard 1–2 hour Kanuma infusion. The FDA label recommends infusion over ≥2 hours; rate may be reduced to no less than 1 hour for stable adults with CESD tolerating maintenance. Sebelipase alfa is enzyme replacement therapy, not chemotherapy — chemo administration codes (96413/96415) are NOT appropriate.

Do I bill JZ or JW for Kanuma?

Both apply on most claims. Kanuma comes only as a 20 mg/10 mL single-dose vial, and dosing is weight-based — virtually every dose produces partial-vial waste. Bill JW with the discarded mg on a separate claim line, AND JZ on the administered mg per CMS's July 2023 single-dose container policy. On Wolman pediatric doses (e.g., a 5 kg infant at 1 mg/kg = 5 mg administered, 15 mg waste), the waste fraction can be three-quarters of the vial — do not omit JW.

Can Kanuma be administered at home?

Home infusion is possible for stable CESD adults with a documented track record of incident-free in-clinic infusions, typically after 6–12 months of supervised therapy. Home infusion is generally NOT appropriate for Wolman infants in early therapy — severe disease, high infusion reaction risk, and the need for emergency response capability mean early Wolman therapy belongs in NICU/PICU or pediatric HOPD.

How is Kanuma billed for a Wolman infant in the NICU or PICU?

Hospital inpatient NICU/PICU administration of Kanuma is bundled into the inpatient DRG / per diem — do not bill J2840 separately on inpatient claims. For outpatient or observation-status NICU/PICU encounters, bill J2840 on the facility outpatient claim (UB-04 / 837I) with the standard JZ + JW modifier pattern. Always verify the encounter type before submitting.

How do I manage Kanuma infusion reactions?

Per FDA label: slow or interrupt the infusion, administer antihistamines, antipyretics, and/or corticosteroids as clinically indicated, and observe the patient until symptoms resolve. For anaphylaxis, stop the infusion immediately and provide standard emergency management. Premedication with antihistamines ± corticosteroids may be appropriate for subsequent infusions in patients with prior reactions. Wolman infants and ADA-positive patients have higher infusion-reaction risk — maintain emergency response capability throughout therapy.

What is a lysosomal storage disorder (LSD)?

Lysosomal storage disorders are a group of ~50–60 inherited metabolic diseases caused by enzyme deficiencies in the lysosome — the cellular organelle responsible for degrading macromolecules. Without the deficient enzyme, undegraded substrate accumulates in tissues, causing progressive multi-organ disease. LAL-D (treated with Kanuma) is one of several LSDs with an FDA-approved recombinant ERT — see also Gaucher disease (Cerezyme, VPRIV, Elelyso), Fabry disease (Fabrazyme, Elfabrio), MPS I (Aldurazyme), MPS II (Elaprase), MPS IVA (Vimizim), MPS VI (Naglazyme), and ASMD (Xenpozyme). Each has a unique HCPCS, NDC, and specific indication — products are not interchangeable.

Source documents

1. DailyMed — KANUMA (sebelipase alfa) Prescribing Information
   FDA-approved label, December 8, 2015 (BLA 125561); current revision
2. AstraZeneca Access 360 — Kanuma HCP reimbursement & coverage materials (formerly Alexion OneSource)
   Patient enrollment, free-drug program, copay assistance, BI/PA support: 1-888-765-4747
3. Kanuma (sebelipase alfa) — patient-facing site (Alexion / AstraZeneca Rare Disease)
4. CMS — Medicare Part B Drug ASP Pricing File
   Q2 2026 quarterly file, effective April 1 – June 30, 2026
5. SEER CanMED — HCPCS J2840 reference
   Permanent code descriptor: "Injection, sebelipase alfa, 1 mg"
6. Burton BK, et al. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency (ARISE). N Engl J Med 2015;373:1010-1020.
   Pivotal CESD trial supporting 1 mg/kg q2w dosing
7. NORD — Lysosomal Acid Lipase Deficiency / LAL-D disease overview
8. UnitedHealthcare — Rare Disease ERT / Kanuma medical drug policy
9. Aetna CPB — Enzyme Replacement Therapy / LAL-D
10. FDA National Drug Code Directory
11. FDA Press Announcement — Kanuma approval (December 8, 2015)

About this page

We maintain this page as a living reference. Medicare ASP pricing is bound to our underlying CareCost data layer and refreshes automatically when CMS publishes new quarterly files. Coding and policy content is reviewed at least quarterly and updated whenever a source document changes.

Found an error? Email hello@carecostestimate.com.

Refresh cadence

Reviewer

Change log

• May 23, 2026 — Boxed Warning correction: Kanuma does carry a BOXED WARNING for HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS per the FDA label (revised July 21, 2025; DailyMed setid 83a77b9d-42f0-4645-8420-786285da7326). Previous version incorrectly stated "no Boxed Warning" in the factcard and the FAQ. Added a prominent BOXED WARNING callout, updated factcard row and FAQ answer, refreshed meta description, and linked the real DailyMed setid.
• May 22, 2026 — Initial publication. ASP data: Q2 2026 (live binding). Manufacturer source: Alexion / AZ Access 360 2025 (post-AstraZeneca acquisition integration). FDA label: current revision (BLA 125561, original approval December 8, 2015). Includes phenotype-specific dosing matrix (Wolman vs CESD), LAL enzyme assay + LIPA genotype PA gate, NICU/PICU billing guidance.

Methodology

Every claim on this page is sourced inline. Pricing reflects the current CMS Part B Drug ASP Pricing File. Payer policies are read directly from each payer's published medical/pharmacy policy documents. Diagnostic and dosing criteria reference the FDA label, the ARISE pivotal trial (NEJM 2015), and NORD / specialist consensus on LAL-D management. We do not paraphrase from billing-software vendor blogs.