CurrentNeighborhood Health Plan of Rhode IslandPolicy Medicaid_Bylvay_Acas.20250205

Medicaid_Bylvay_Acas.20250205

Defines medical necessity criteria, exclusions, authorization durations, continuation criteria, dosing limits, and quantity limits for Bylvay (odevixibat) for treatment of pruritus in PFIC and cholestatic pruritus in Alagille syndrome for Medicaid members.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyMedicaid_Bylvay_Acas.20250205

Policy CodePolicy Medicaid_Bylvay_Acas.20250205

Change TypeReviewed / revised (no material clinical change)

Effective DateApr 1, 2022

Next Review Date

Key ActionProviders must submit prior authorization with evidence of diagnosis, molecular testing results, documentation of moderate to severe pruritus with drug-induced causes ruled out, prior treatment history, current weight and prescribed dose, and lab evidence of cholestasis as specified.

SourceLink

POLICY UPDATE CHANGES

Reviewed on multiple dates through 02/2025 with policy content current as of 02/2025.

2Covered Indications

6 monthsAuth duration (initial/renewal)

<=120 mcg/kg/dayMax dose allowed

>=100 µmol/LPFIC bile acid threshold

Coverage Summary

Bylvay (odevixibat) is covered with criteria for the FDA‑approved indications: treatment of pruritus in progressive familial intrahepatic cholestasis (PFIC) for patients ≥ 3 months of age and treatment of cholestatic pruritus in Alagille syndrome (ALGS) for patients ≥ 12 months. Coverage requires molecular or clinical diagnostic documentation and laboratory thresholds of cholestasis as specified for each indication, and the medication must be prescribed by or in consultation with a hepatologist, gastroenterologist, or a clinician who specializes in the relevant disorder. Initial and continuation authorizations are generally limited to 6 months and require documentation of benefit for renewal. Dosing limits are weight‑based with PFIC initial dosing not to exceed 40 mcg/kg/day (with escalation to a maximum of 120 mcg/kg/day under specified conditions) and ALGS dosing not to exceed 120 mcg/kg/day; PFIC also has an absolute maximum dose of 6 mg/day.

Initial Therapy Criteria

FDA-Approved Indications - Covered when ALL criteria met

The FDA-approved indications below are covered provided all approval criteria are met and no exclusions apply. Initial PFIC authorization limited to 6 months.

ALL of the following

Indication: pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC).
Member is 3 months of age or older.
Medication prescribed by or in consultation with a hepatologist or gastroenterologist or a physician that specializes in pruritus in PFIC.
Documentation that the member has moderate to severe pruritus and drug-induced pruritus has been ruled out.
Member has a confirmed molecular diagnosis of PFIC (e.g., mutations in ATP8B1, ABCB11, TJP2, MYO5B, and ABCB4).
Documentation that the member has serum bile acid level ≥ 100 µmol/L.>= 100 µmol/L
Documentation that the member does not have any other concomitant liver disease (e.g., cirrhosis, biliary atresia, benign recurrent intrahepatic cholestasis [BRIC], liver cancer, alternate non-PFIC related etiology of cholestasis) or history of a hepatic decompensation event (e.g., variceal hemorrhage, ascites, hepatic encephalopathy, portal hypertension).
Documentation that the member has not received a liver transplant or surgical interruption of the enterohepatic circulation (e.g., partial external biliary diversion surgery).
Documentation that the member experienced an inadequate treatment response or intolerance to at least two systemic medications for PFIC-related pruritus (e.g., ursodiol 20-30 mg/kg/day, rifampin, cholestyramine).
Documentation that the member's dose will not exceed 40 mcg/kg/day for PFIC initial authorization. Member's current weight and prescribed dose must be provided.<= 40 mcg/kg/day
Initial PFIC authorization limited to 6 months

FDA-Approved Indication - Alagille syndrome (ALGS) Covered when ALL criteria met

Authorization of 6 months may be granted for treatment of cholestatic pruritus in ALGS when all criteria below are met. Initial ALGS authorization limited to 6 months.

ALL of the following

Indication: cholestatic pruritus in patients 12 months of age and older with Alagille syndrome (ALGS).
Member is 12 months of age or older.
Medication prescribed by or in consultation with a hepatologist or gastroenterologist or a physician that specializes in Alagille syndrome.
Diagnosis established by ONE of
- Genetic testing (mutations in JAG1 or NOTCH2).

Continuation Therapy Criteria

Continuation of Therapy - Covered when ALL criteria met

Authorization of 6 months may be granted for continuation when request includes documentation meeting all criteria below.

ALL of the following

The member meets all initial criteria.
The member is experiencing benefit from therapy (e.g., improvement in pruritus and reduction in serum bile acid).
Member's dose will not exceed 120 mcg/kg/day and if requesting dose increase for PFIC, documentation supports no improvement in pruritus after at least 3 months at each dose of 40 mcg/kg/day and 80 mcg/kg/day, if applicable.<= 120 mcg/kg/day

Unproven / Exclusions & Thresholds

Explicit exclusions and limits include: exclusion of members with PFIC type 2 who have ABCB11 gene variants that predict nonfunctional or complete absence of the bile salt export pump protein (designated BSEP-3). Coverage is also not provided when the member has other concomitant liver disease (for example cirrhosis, biliary atresia, BRIC, liver cancer) or a history of hepatic decompensation, or when the member has previously received a liver transplant or surgical interruption of the enterohepatic circulation (e.g., partial external biliary diversion). Numeric dosing thresholds include a PFIC initial dosing cap of 40 mcg/kg/day, allowance to escalate up to 120 mcg/kg/day in PFIC only after specified trial periods, and a PFIC maximum absolute dose of 6 mg/day.

Numeric thresholds / dosing limits

PFIC serum bile acid threshold>= 100 µmol/L

ALGS total serum bile acid threshold> 3 x ULN for age

ALGS conjugated bilirubin threshold> 1 mg/dL

ALGS GGT threshold> 3 x ULN for age

PFIC initial dose limit<= 40 mcg/kg/day

ALGS and continuation maximum dose<= 120 mcg/kg/day

PFIC maximum absolute dose6 mg/day

Provider Actions & Billing Rules

Prior Authorization

Prior authorization required with documentation

Submit a prior authorization with: diagnosis (PFIC or ALGS indication); molecular testing results confirming the PFIC or ALGS genotype; documentation of moderate-to-severe pruritus with drug-induced causes ruled out; prior treatment history showing inadequate response or intolerance to at least two systemic therapies for pruritus; the member's current weight and the prescribed dose; and laboratory evidence of cholestasis as specified in the policy.

Diagnosis: PFIC or ALGS (per indications)
Molecular testing results (genotype for PFIC or ALGS)
Documentation of moderate–severe pruritus and exclusion of drug‑induced pruritus
Prior treatment history: inadequate response/intolerance to ≥2 systemic therapies
Current weight and prescribed dose (to confirm mcg/kg/day limits)
Lab evidence of cholestasis (e.g., serum bile acids, conjugated bilirubin, GGT, vitamin deficiency) as applicable

Documentation Required

Exclude BSEP-3 ABCB11 variants for PFIC type 2

For PFIC, the policy excludes patients with ABCB11 variants that predict nonfunctional or absent BSEP protein (BSEP‑3). Provide genotype documentation to demonstrate the member does not have BSEP‑3 ABCB11 variants to avoid exclusion.

Billing Rule

Quantity limits per formulation

Adhere to the listed quantity limits for each Bylvay formulation when submitting claims; use the specified product formulation and corresponding 30‑day quantity limit.

Bylvay oral pellets 200 mcg — 360 per 30 days
Bylvay oral pellets 600 mcg — 120 per 30 days
Bylvay capsules 400 mcg — 540 per 30 days
Bylvay capsules 1200 mcg — 180 per 30 days

Documentation Required

Dose and weight documentation for dosing limits

Provide the member's current weight and the prescribed dose with the request so dosing can be confirmed not to exceed the policy mcg/kg/day limits (PFIC initial limit 40 mcg/kg/day; ALGS and continuation limit up to 120 mcg/kg/day).

Denial Risk

Denial for excluded genotype or alternate liver disease

Claims will be denied if the member meets exclusionary conditions: PFIC type 2 with ABCB11 variants predicting nonfunctional or absent BSEP protein (BSEP‑3), presence of other concomitant liver disease (e.g., cirrhosis, biliary atresia, liver cancer) or history of hepatic decompensation, or prior liver transplant or surgical interruption of enterohepatic circulation (e.g., partial external biliary diversion).

Background & Definitions

FDA approval summary from the brief: Bylvay is indicated for pruritus in PFIC for patients ≥ 3 months of age and for cholestatic pruritus in ALGS for patients ≥ 12 months. A specialist (hepatologist, gastroenterologist, or clinician experienced in the condition) must prescribe or consult on therapy. The policy explicitly excludes use in PFIC type 2 when ABCB11 variants predict absent or nonfunctional BSEP protein.

Term	Definition
BSEP-3	ABCB11 gene variants predicting nonfunctional or complete absence of bile salt export pump protein.
ALGS major clinical features	Hepatic abnormality; cardiac abnormality; skeletal abnormality; ophthalmologic abnormality; characteristic facial features; vascular abnormalities; renal structural/functional abnormality.