CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Pombiliti (cipaglucosidase alfa-atga) with Opfolda (miglustat) for late-onset Pompe disease

Coverage policy for use of Pombiliti in combination with Opfolda to treat adult patients with late-onset Pompe disease for Neighborhood Health Plan of Rhode Island members (Medicaid, Commercial, Medicare-Medicaid Plan).

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyPombiliti (cipaglucosidase alfa-atga) with Opfolda (miglustat) for late-onset Pompe disease

Policy CodePolicy N/A

Change TypeNo material change

Effective DateJul 1, 2024

Next Review DateMay 8, 2024

Key ActionSubmit prior authorization with baseline FVC and 6MWT values and documentation of inadequate response or intolerance to prior ERT.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsinitial authorization length

20 mg/kg q14dPombiliti dosing

260/195 mgOpfolda dosing

J1203, J1202HCPCS codes listed

E74.02Covered diagnosis code

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Coverage Criteria for Pombiliti + Opfolda

inv-01: Initial Approval Criteria

Covered when ALL of the following are met

Age: Patient is at least 18 years of age>=18 years

Clinical signs: Patient has measurable signs of Pompe disease such as impairment in pulmonary function or motor weakness

Baseline testing: Documented baseline percent predicted FVC >=30% and 6MWT distance >=75 meters are submittedFVC >=30%; 6MWT >=75 m

Submit baseline values

Reproductive precautions: Females of reproductive potential have a negative pregnancy test prior to initiating therapy and will use effective contraception during treatment

Prior ERT response: Patient is experiencing an inadequate response or intolerance to current enzyme replacement therapy (e.g., alglucosidase-alfa, avalglucosidase-alfa)

Defined as not improving on current ERT

No combination ERT: Will not be used in combination with other enzyme replacement therapies (e.g., alglucosidase-alfa, avalglucosidase-alfa)

Combination with Opfolda: Pombiliti must be used in combination with the enzyme stabilizer miglustat (Opfolda) per dosing/administration instructions

If Opfolda dose missed, Pombiliti should not be given; initiate Pombiliti approximately 1 hour after Opfolda

Weight requirement: Patient has an actual body weight of at least 40 kilograms>=40 kg

Pombiliti dosing is weight-based (20 mg/kg)

Ventilation support: Patient does not require invasive or noninvasive ventilation support for more than 6 hours per day while awake<=6 hours/day

Diagnosis confirmation: Diagnosis confirmed by EITHER deficiency of acid alpha-glucosidase (GAA) enzyme activity OR detection of biallelic pathogenic variants in the GAA gene by molecular genetic testing

Patient has diagnosis of late-onset (non-infantile) disease

inv-02: Renewal Criteria

Renewal covered when ALL of the following are met

Continuation of criteria: Patient continues to meet universal and other indication-specific relevant criteria (e.g., concomitant therapy requirements, performance status)

Toxicity: Absence of unacceptable toxicity from the drug (examples: anaphylaxis, severe hypersensitivity, severe infusion-associated reactions, acute cardiorespiratory failure)

Clinical benefit: Patient has demonstrated beneficial response compared to pretreatment baseline in stabilization or improvement in percent predicted FVC and/or 6-minute walk test (6MWT)

Antibody monitoring: Patient is being monitored for antibody formation, including neutralizing antibodies

Use in combination with other enzyme replacement therapies is excluded. Pombiliti must not be administered concurrently with other ERTs such as alglucosidase-alfa (Lumizyme) or avalglucosidase-alfa (Nexviazyme).

Requests for Pombiliti will not be supported when patients do not meet the policy's minimum eligibility thresholds. Specifically, coverage is not supported for patients who are <18 years of age, weigh <40 kg, or lack documented baseline pulmonary and functional testing demonstrating percent predicted FVC ≥30% and 6MWT distance ≥75 meters. Continued or concomitant use with other enzyme replacement therapies is likewise not supported.

inv-16: Diagnostic confirmation

Molecular confirmation: Detection of biallelic pathogenic variants in the GAA gene by molecular genetic testing establishes the diagnosis of late-onset Pompe disease

Molecular genetic testing is an accepted method to confirm diagnosis

Coding and Clinical Thresholds

HCPCS CodesHCPCSCovered

J1203	Injection, cipaglucosidase alfa-atga, 5 mg
J1202	Miglustat, oral, 65 mg

NDCNDC

71904-0200-xx	Pombiliti 105 mg single-dose vial NDC
71904-0300-xx	Opfolda 65 mg oral capsule NDC

Covered Diagnosis CodesICD-10Covered

E74.02

Pompe disease

inv-08: 6-Minute Walk Test (6MWT) distance — clinical threshold

6MWT threshold>= 75 meters

ContextDocumented baseline 6-minute walk test (6MWT) distance must be submitted as part of initial approval.

Use in renewalRenewal requires demonstration of stabilization or improvement in 6MWT compared to pretreatment baseline.

inv-09: Percent predicted forced vital capacity (FVC) — clinical threshold

Percent predicted FVC threshold>= 30% of predicted

ContextDocumented baseline percent predicted forced vital capacity (FVC) must be submitted as part of initial approval.

Provider Requirements and Billing

Prior Authorization

Prior Authorization Required

Prior authorization required. Initial approval is provided for 6 months and may be renewed for additional 6‑month periods.

Applies to claims billed with HCPCS J1203 (cipaglucosidase alfa-atga, 5 mg).
Initial approval: 6 months; renewals may be granted for 6 months.

Step Therapy

Prerequisite Therapy

Coverage requires prior inadequate response or intolerance to current enzyme replacement therapy before initiating cipaglucosidase alfa.

Prerequisite therapies include alglucosidase alfa (Lumizyme) or avalglucosidase alfa (Nexviazyme).

Member Eligibility Requirements

Coverage is provided when ALL of the following top-level eligibility requirements are met: patient is at least 18 years old; has measurable signs of Pompe disease (pulmonary impairment or motor weakness); baseline testing shows FVC ≥30% predicted and 6MWT ≥75 meters; females of reproductive potential have a negative pregnancy test and will use effective contraception during treatment; patient has had an inadequate response or intolerance to current ERT; Pombiliti will not be used in combination with other ERTs; Pombiliti will be administered with miglustat (Opfolda) per dosing instructions; patient has an actual body weight of at least 40 kg; and patient does not require invasive or noninvasive ventilation for >6 hours per day while awake.

Definitions

inv-15: LOPD definition — non-infantile acid alpha-glucosidase (GAA) deficiency diagnosed by GAA enzyme deficiency or biallelic pathogenic GAA variants

DefinitionLate-onset Pompe disease (LOPD) is non-infantile acid alpha-glucosidase (GAA) deficiency.

Diagnostic criteriaDiagnosis confirmed by EITHER deficiency of GAA enzyme activity OR detection of biallelic pathogenic variants in the GAA gene by molecular genetic testing.

Indication contextPolicy applies to adult patients with late-onset (non-infantile) disease weighing >= 40 kg who are not improving on current ERT.

Background

Late-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency) causes progressive respiratory and motor weakness. Pombiliti (cipaglucosidase alfa-atga) is indicated for adult patients with late-onset Pompe disease and must be given in combination with the enzyme stabilizer miglustat (Opfolda). Clinical data demonstrated numerically favorable but not statistically superior primary endpoint results; the therapy carries warnings for severe hypersensitivity, infusion reactions, and risk of acute cardiorespiratory failure, and patients with compromised cardiac or respiratory function should be closely monitored during infusion.