CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

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

Clinical coverage criteria for use of Pombiliti in combination with Opfolda for adult patients with late-onset Pompe disease (LOPD). Applies to Medicaid, Commercial, and Medicare-Medicaid Plan members of Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

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

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJul 1, 2024

Next Review DateN/A

Key ActionSubmit prior authorization with baseline FVC percent predicted and 6MWT distance, documentation of inadequate response or intolerance to prior ERT, diagnostic confirmation, weight, pregnancy test for females of reproductive potential, and prior treatment history.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsInitial authorization length

20 mg/kg q14 daysPombiliti dosing

260 mg or 195 mgOpfolda dosing (weight-based)

≥40 kgMinimum patient weight for therapy

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Initial Approval Criteria

Coverage is provided when ALL of the following are met:

(All criteria above must be met for initial coverage)

Renewal Criteria

Coverage may be renewed when ALL of the following are met:

ALL of the following

Patient continues to meet universal and other indication-specific criteria (e.g., concomitant therapy requirements, performance status).
Absence of unacceptable toxicity from the drug (examples: anaphylaxis, severe hypersensitivity, severe infusion-associated reactions, acute cardiorespiratory failure).
Patient has demonstrated beneficial response compared to pretreatment baseline in one or more measures: stabilization or improvement in FVC and/or 6MWT.
Patient is being monitored for antibody formation (including neutralizing antibodies).

(Renewal requests should include documentation of clinical response measures and antibody monitoring)

Length of Authorization and Quantity Limits

Operational limits and dispensing rules

ALL of the following

Initial and renewal authorizations: 6 months each.
Coverage will be initially provided for 6 months and may be renewed for 6 months.
- Pombiliti 105 mg single-dose vial: 22 vials every 14 days.
  NDC unit limit
- Opfolda 65 mg oral capsules: 4 capsules every 14 days.
  NDC unit limit (260 mg total)
- Pombiliti: 2300 mg every 14 days.

Authorization, Documentation, and Denial Risks

Prior Authorization

Prior authorization required with documentation

Prior authorization required. Submit baseline percent predicted FVC and 6-minute walk test (6MWT) distance, evidence of inadequate response or intolerance to prior enzyme replacement therapy (ERT), diagnostic confirmation of Pompe disease (GAA enzyme deficiency or biallelic pathogenic GAA variants), patient weight, pregnancy test for females of reproductive potential, and prior treatment history. Applicable codes: E74.02, J1203, J1202.

Baseline percent predicted FVC (≥30% of predicted required)
Baseline 6MWT distance (≥75 meters required)
Evidence of inadequate response or intolerance to prior ERT (e.g., alglucosidase-alfa, avalglucosidase-alfa)
Diagnostic confirmation: GAA enzyme deficiency OR biallelic pathogenic GAA variants
Actual body weight (must be ≥40 kg)
Pregnancy test for females of reproductive potential
Prior treatment history (including prior ERT duration)

Documentation Required

Monitoring and documentation during therapy

Document monitoring for antibody formation, including neutralizing antibodies, and record clinical response for renewal requests; renewal requires stabilization or improvement in FVC and/or 6MWT compared to pretreatment baseline.

Report antibody monitoring results (including neutralizing antibodies) at renewal
Document clinical measures showing stabilization or improvement in FVC and/or 6MWT

Denial Risk

Denial risk for concurrent ERT or missed Opfolda dosing

Requests will be denied if Pombiliti is used concurrently with other ERTs or if Opfolda is not used as required; Pombiliti should not be administered if the Opfolda dose was missed.

Affected codes: J1203 (cipaglucosidase alfa-atga) and J1202 (miglustat)
Denial conditions: concurrent use with other ERTs (e.g., Lumizyme, Nexviazyme)
Denial condition: Opfolda not used per dosing guidance or Opfolda dose missed (do not administer Pombiliti if Opfolda dose was missed)

Documentation Required

Pregnancy and contraception requirement

Obtain a negative pregnancy test before initiating therapy in females of reproductive potential and document a plan to use effective contraception during treatment.

Negative pregnancy test required prior to initiation
Document planned effective contraception for duration of treatment

Clinical Thresholds, Dosing, and Quantity Limits

Therapy thresholds and limits

Minimum age≥ 18 years

Minimum weight≥ 40 kg

FVC baseline≥ 30% predicted

6MWT baseline distance≥ 75 meters

Pombiliti dose20 mg/kg every 14 days (IV infusion ≈4 hours)

Opfolda dosing260 mg for ≥50 kg; 195 mg for ≥40–<50 kg; initiate ~1 hour before infusion

Quantity limitsPombiliti: 22 vials every 14 days (105 mg vials); Opfolda: 4 capsules (65 mg) every 14 days (260 mg)

6 monthsInitial authorization length

20 mg/kg q14 daysPombiliti dosing

260 mg / 195 mgOpfolda dosing (weight-based)

>=40 kgMinimum patient weight for therapy

Clinical Background

Pombiliti (cipaglucosidase alfa-atga) administered in combination with the oral enzyme stabilizer Opfolda (miglustat) is indicated for adult patients with late-onset Pompe disease (LOPD) weighing ≥ 40 kg who are not improving on their current enzyme replacement therapy (ERT). The indication and trial evidence come from a randomized, double-blind, active-controlled phase 3 trial (PROPEL) in which 123 adults were randomized 2:1 to receive Pombiliti plus Opfolda or an alglucosidase alfa product for 52 weeks; about 77% of participants were ERT experienced (defined as ≥2 years, mean ~7.4 years prior ERT exposure).

In the PROPEL trial the primary outcome—change in 6-minute walk test (6MWT) distance compared with baseline—favored the Pombiliti plus Opfolda arm numerically, but the difference was not determined to be statistically superior. Clinical use therefore emphasizes documented baseline measures and demonstration of stabilization or improvement in functional measures (e.g., percent predicted forced vital capacity [FVC] and 6MWT) when evaluating benefit.

Clinically important safety considerations include a Boxed Warning for severe hypersensitivity reactions, infusion-associated reactions, and risk of acute cardiorespiratory failure in susceptible patients. Infusions may require pretreatment with antihistamines, antipyretics, and/or corticosteroids, and patients with fluid overload risk or compromised cardiac/respiratory status should be closely monitored during infusion.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

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

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJul 1, 2024

Next Review DateN/A

SourceLink

On This Page

71904-0200-xx	Pombiliti 105 mg single-dose vial powder for injection
71904-0300-xx	Opfolda 65 mg oral capsules

OpenPayer

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

Medical Necessity Criteria

Relevant Codes

Authorization, Documentation, and Denial Risks

Clinical Thresholds, Dosing, and Quantity Limits

Clinical Background

Defined Terms

Policy Dates & Revision Notes

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