ModifiedCentenePolicy CP PHAR.45

Voxelotor (Oxbryta)

Centene clinical policy governing medical necessity, continuation criteria, dosing, and billing-relevant rules for voxelotor (Oxbryta) across Commercial, HIM, and Medicaid lines of business; includes manufacturer market withdrawal and related continued-therapy guidance.

Policy Summary

PayerCentene

PolicyVoxelotor (Oxbryta)

Policy CodePolicy CP PHAR.45

Change TypeManufacturer market withdrawalcontinued-therapy criteria revised

Effective Date03.01.20

Next Review Date

Key ActionProvider must submit documentation supporting criteria, attest to awareness of Pfizer's market withdrawal, and collaborate with the member on next treatment steps; initial starts are not permitted.

SourceLink

POLICY UPDATE CHANGES

Initial approval criteria removed due to manufacturer withdrawal of Oxbryta.

Added provider attestation acknowledging market withdrawal and collaboration on next steps as a continued-therapy criterion.

Revised continued therapy approval duration to 1 month.

Updated maximum dosing requirements to allow dose adjustments for CYP3A4 inducers (strong and moderate).

Appendix E added documenting Pfizer voluntary market withdrawal of Oxbryta (September 25, 2024).

1Approval duration (months) for continuation

>=1 g/dLHemoglobin response required

0New starts allowed

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2024-09-25Manufacturer withdrawal date

Coverage Summary & Scope

Policy CP PHAR.45 addresses Voxelotor (Oxbryta) — a hemoglobin S polymerization inhibitor — for treatment of sickle cell disease (SCD) in patients aged ≥4 years. The Centene policy covers Commercial, HIM, and Medicaid lines of business and governs medical necessity, continuation criteria, dosing, and billing rules. The indication was originally granted under accelerated approval based on an increase in hemoglobin. The manufacturer, Pfizer, voluntarily withdrew Oxbryta for SCD on 2024-09-25, and the policy was modified accordingly (initial approval criteria removed; continued-therapy provisions revised). Effective date: 03.01.20; Last review date: 02.25.

Initial Therapy Criteria

Initial Approval Criteria

Covered when ALL of the following are met:

ALL of the following

ALL of the following
- Authorization is not permitted; member may not initiate therapy with Oxbryta.
- If member is currently using Oxbryta, proceed to Continued Therapy section.
- Diagnosis: Sickle Cell Disease (SCD).

Continuation Therapy (Maintenance) Criteria

Continued Therapy (Maintenance) Criteria

Covered when ALL of the following are met:

ALL of the following

Member meets one of the following:
- Currently receiving medication via Centene benefit.
- Member has previously met initial approval criteria.
- Member is currently receiving medication and is enrolled in a state/product with continuity of care regulations (refer to state addendums).
Diagnosis: Sickle Cell Disease (must meet all diagnosis requirements).

Dosing / Maximum Dose Constraints

Dosing / Maximum Dose Constraints (Decision Logic)

Dose maxima depend on CYP3A4 inducer co-medication status:

ALL of the following

If member is concurrently taking a strong CYP3A4 inducer
- Maximum dose for adults and children >= 40 kg: 2,500 mg (5 tablets) per day.
- Maximum dose for children 20–40 kg: 1,500 mg per day when strong inducer (per dosing table).
- Maximum dose for children 10–20 kg: 900 mg per day when strong inducer (per dosing table).
If member is concurrently taking a moderate CYP3A4 inducer

Diagnoses / Indications Not Authorized

Diagnoses/Indications Not Authorized

ANY of the following

Non-FDA approved indications which are not addressed in this policy, unless sufficient documentation of efficacy and safety per off-label use policies (CP.CPA.09, HIM.PA.154, CP.PMN.53) or other evidence of coverage.

Applicable Codes

Supporting ICD-10-CM Diagnosis CodesICD-10Covered

D57.0	Hb-SS disease with crisis
D57.1	Sickle-cell disease without crisis
D57.2	Sickle-cell/Hb-C disease
D57.4	Sickle-cell thalassemia

Product NDC / HCPCS (not specified in policy)mixed

No codes listed

Provider Actions & Authorization Requirements

Prior Authorization

Prior authorization required for continued therapy

Provider must submit documentation (such as chart notes, lab results or other clinical information) supporting that member has met all approval criteria; initial authorization for new starts is not permitted.

Documentation Required

Documentation of hemoglobin response

Provider must document an increase in hemoglobin (Hb) from baseline of at least 1 g/dL to support continued therapy.

Documentation Required

Provider attestation of market withdrawal

Provider must attest they acknowledge Pfizer's voluntary market withdrawal of Oxbryta and that they are actively collaborating with the member to establish next treatment steps.

Background & Clinical Evidence

Voxelotor is a hemoglobin S (HbS) polymerization inhibitor indicated for treatment of sickle cell disease in adults and pediatric patients ≥4 years. The indication received accelerated approval based on demonstration of an increase in hemoglobin (Hb); continued approval may be contingent on confirmatory trials. Pfizer announced a voluntary global withdrawal of Oxbryta for SCD on September 25, 2024, citing that the totality of clinical data no longer supported a favorable benefit–risk profile and noting concerns about imbalances in vaso-occlusive crisis (VOC) and fatal events; Pfizer also discontinued active trials and expanded access programs and advised that patients should contact their physicians. The policy cites the key Phase 3 randomized trial by Vichinsky et al. (N Engl J Med, 2019) as supporting evidence for the approval basis.

Evidence / Regulatory and Trial Highlights

Regulatory statusAccelerated approval for SCD; continued approval contingent on confirmatory trials; manufacturer voluntary withdrawal announced 2024-09-25 (patients should no longer be prescribed Oxbryta)

Key trialPhase 3 randomized trial (Vichinsky et al., N Engl J Med. 2019)

Required hemoglobin response for continuation>= 1 g/dL increase from baseline

Approval duration for continued therapy

Definitions

Hb: Hemoglobin — the oxygen-carrying protein in red blood cells.

SCD: Sickle Cell Disease — inherited hemoglobin disorder characterized by sickling of red blood cells.

VOC: Vaso-occlusive crisis — acute painful episodes due to microvascular occlusion in SCD.

Revision History

2024-09-25added

Appendix E added documenting Pfizer voluntary market withdrawal of Oxbryta (September 25, 2024); describes withdrawal rationale, advises patients should no longer be prescribed Oxbryta, recommends contacting physicians, and notes lack of established tapering guidance.

2024-10-08removed

Initial approval criteria removed due to manufacturer withdrawal of Oxbryta (RT4 removed initial approval criteria).

2024-10-08added

Policy Summary

PayerCentene

PolicyVoxelotor (Oxbryta)

Policy CodePolicy CP PHAR.45

Change TypeManufacturer market withdrawalcontinued-therapy criteria revised

Effective Date03.01.20

Next Review Date

SourceLink

On This Page

Member is responding positively to therapy as evidenced by an increase in hemoglobin (Hb) level from baseline of at least 1 g/dL.>= 1 g/dL increase from baseline

Oxbryta is prescribed concurrently with hydroxyurea, unless contraindicated or clinically significant adverse effects are experienced.

Provider attestation acknowledging market withdrawal of Oxbryta and that provider is actively collaborating with the member to establish next steps in the treatment plan.

Oxbryta is not prescribed concurrently with Adakveo (crizanlizumab).

Dosing limits: If request is for a dose increase, new dose does not exceed the applicable maximum based on concomitant CYP3A4 inducer status and age/weight per the dosing table.

Approval duration is 1 month.

Maximum dose for adults and children >= 40 kg: 2,000 mg (4 tablets) per day.

Maximum dose for children 20–40 kg: 1,200 mg per day when moderate inducer.

Maximum dose for children 10–20 kg: 900 mg per day when moderate inducer.

For all other members (no relevant inducer)

Maximum dose for adults and children >= 40 kg: 1,500 mg (3 tablets) per day.
For children 20–40 kg: 900 mg PO QD.
For children 10–20 kg: 600 mg PO QD.

Billing Rule

Approval duration for continuation

Continued therapy approvals are issued for 1 month.

Denial Risk

Initiation requests will be denied

Authorization is not permitted for members initiating Oxbryta; requests to start new therapy will be denied per policy.

Documentation Required

Concomitant therapy constraints

Oxbryta should not be prescribed concurrently with Adakveo (crizanlizumab); concurrent use with hydroxyurea is expected unless contraindicated or clinically significant adverse effects are experienced.

Documentation Required

Dose change documentation

For dose increase requests, provider must document that the new dose does not exceed the applicable maximum based on concomitant CYP3A4 inducer status and age/weight per the dosing table.

Maximum adult dose (no inducer)1,500 mg PO QD; with moderate CYP3A4 inducer: 2,000 mg PO QD; with strong inducer: 2,500 mg PO QD

Manufacturer guidance on discontinuationPfizer advised patients should contact physicians; no established tapering dose or regimen for gradual discontinuation

Added provider attestation requirement as a continued-therapy criterion: provider must acknowledge market withdrawal and attest they are actively collaborating with the member to establish next treatment steps.

2024-10-08revised

Revised continued therapy approval duration to 1 month (continued therapy approvals are issued for 1 month).

2023-02-23revised

Updated maximum dosing requirements to allow dose adjustments for CYP3A4 inducers (strong and moderate) with specific maxima by age/weight and inducer status reflected in the dosing table.

2025-02-25noteLatest

Policy last reviewed and remains modified to reflect the market withdrawal and related continued-therapy guidance (policy status: MODIFIED).