CurrentBlue Cross Blue Shield - IowaPolicy 05.04.68

Vijoice (alpelisib) for PIK3CA-Related Overgrowth Spectrum (PROS)

Defines prior authorization, coverage criteria, continuation criteria, dosing/quantity limits, and documentation requirements for Vijoice (alpelisib) to treat PROS in adult and pediatric patients.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyVijoice (alpelisib) for PIK3CA-Related Overgrowth Spectrum (PROS)

Policy CodePolicy 05.04.68

Change TypeRevised (June 2024); Reviewed (Jan 2026)

Effective DateJun 28, 2024

Next Review Date

Key ActionSubmit documentation confirming PIK3CA mutation for initial requests and chart notes/medical records demonstrating positive clinical response for continuation requests; initial approvals for 6 months and continuation approvals for 12 months.

POLICY UPDATE CHANGES

Policy revised June 2024; current effective date June 28, 2024.

Policy reviewed January 2026.

1Covered Indication (PROS)

6/12Approval durations (months): initial/continuation

56Tablet qty per 28 days

28Oral granules pkts per 28 days

Coverage Summary

Vijoice (alpelisib) is FDA-approved to treat adults and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. This policy defines prior authorization, coverage criteria, continuation criteria, dosing/quantity limits, and documentation requirements for Vijoice (alpelisib) to treat PROS in adult and pediatric patients. Coverage stance: covered_with_criteria. Policy number: 05.04.68. Effective date: 2024-06-28.

Quick facts and thresholds

Minimum age>= 2 years

Initial approval duration6 months

Continuation approval duration12 months

Tablet quantity limit56 tablets per 28 days

Oral granules quantity limit28 packets per 28 days

Initial Therapy Criteria

Initial Authorization - PROS

Authorization of 6 months may be granted...

ALL of the following

Member is at least 2 years of age
Member has severe manifestations of disease and requires systemic therapy
Documentation of test confirming presence of PIK3CA mutation
Submission required to initiate prior authorization review

Continuation Therapy Criteria

Continuation of Therapy

Authorization of 12 months may be granted...

ALL of the following

All initial criteria are met
No evidence of unacceptable toxicity while on the current regimen
No evidence of disease progression while on the current regimen
Chart notes or medical record documentation supporting positive clinical response
Submission required for continuation requests

Not Medically Necessary / Exclusions

Not Medically Necessary

Vijoice is considered not medically necessary when the following applies:

ANY of the following

Member does not meet the criteria set forth for initial or continuation therapy

Provider Actions & Requirements

Prior Authorization

Prior authorization required for initial and continuation therapy

Prior authorization is required for both initial and continuation therapy with Vijoice (alpelisib). Initial approvals may be granted for 6 months; continuation approvals may be granted for 12 months.

Initial approvals = 6 months
Continuation approvals = 12 months

Documentation Required

Required documentation for review

Submit required documentation to support review. For initial requests, provide documentation of a test confirming presence of a PIK3CA mutation. For continuation requests, provide chart notes or medical record documentation supporting positive clinical response. These submission requirements are referenced in the criteria for initial and continuation authorization.

Initial requests: PIK3CA mutation test results
Continuation requests: chart notes/medical records showing positive clinical response

Billing Rule

Quantity limits enforced

Quantity limits are enforced for Vijoice product forms per the policy.

Tablets - 56 tablets per 28 days
Oral granules - 28 packets per 28 days

Clinical Evidence

27%Radiologic response rate at week 24 (≥20% reduction in sum of target lesion volume)

60%Duration of response ≥12 months among responders

14%Mean reduction in sum of target lesion volume among imaged patients

Key evidence sources supporting the approval and use of Vijoice for PROS include the EPIK-P1 retrospective chart review (compassionate use program) which reported a 27% radiological response rate at week 24 and a 60% duration of response ≥12 months, with reductions in target lesion volume and generally manageable safety findings, and the FDA approval documentation (Vijoice package insert, April 2024). Together these data supported the FDA approval of Vijoice for patients with severe PROS requiring systemic therapy.

Background & Definitions

PROS (PIK3CA-Related Overgrowth Spectrum) is a group of rare genetic disorders caused by activating PIK3CA mutations that lead to asymmetric regional overgrowth of various tissues (e.g., fat, muscle, bone, nerve, brain, and blood vessels) affecting areas such as the brain, limbs, trunk, and face. Vijoice (alpelisib) is a targeted PIK3CA inhibitor indicated for patients aged 2 years and older with severe manifestations of PROS who require systemic therapy; it inhibits the PIK3CA-driven signaling pathway implicated in these overgrowth disorders. Traditional management of PROS is primarily supportive and surgical (including surgery, sclerotherapy, and psychological and nutritional support), and some patients have received mTOR inhibitors (e.g., sirolimus) with moderate and inconsistent effects; targeted PIK3CA inhibition with alpelisib offers an approved therapeutic option for severe disease. Quantity limits are specified for dispensing: 56 tablets per 28 days for tablets and 28 packets per 28 days for oral granules.

PROS: PIK3CA-Related Overgrowth Spectrum is a group of genetic disorders causing regional overgrowth due to activating PIK3CA mutations.