Diagnosis: Pompe Disease.
Treatment: Lumizyme (dosed at 40mg (milligrams)/kg (kilograms) weekly.

The insurer denied: Lumizyme (dosed at 40mg (milligrams)/kg (kilograms) weekly.
The denial is overturned.

The patient is an adult male with atypical infantile Pompe disease. Treatment with Lumizyme 40 mg/kg (milligrams/kilogram) is the subject of this review. The drug was denied coverage by the health plan based on the requested dose. The provider appealed the determination. The provider indicates that the patient is currently receiving 47 mg/kg (milligrams/kilogram) every other week although the plan indicates that the patient has only been approved for 20 mg/kg (milligrams/kilogram) every other week.

The health plan should cover the requested Lumizyme.

Although the requested dose is not supported by the FDA (United States Food and Drug Administration) prescribing information, the peer reviewed literature suggests that there are improved outcomes for patients treated with higher doses of this medication and more frequently.

The requested Lumizyme is more beneficial.

The request is for Lumizyme 40 mg/kg (milligrams per kilogram) weekly. The FDA (United States Food and Drug Administration) and manufacturer approved dosing is 20 mg/kg (milligrams per kilogram) every other week. The physician has submitted literature for the increased dose of 40 mg/kg (milligrams per kilogram) per week from an open label single-center study that concluded the higher dose of 40 mg/kg (milligrams per kilogram) per week improves outcome measures. This finding was repeating in a larger cohort study that showed improved outcomes with higher dosing. Patients with infantile Pompe disease treated with the high enzyme replacement therapy dosage of 40 mg/kg (milligrams per kilogram) per week had significantly improved survival when compared with patients treated with the standard recommended enzyme replacement therapy dosage of 20 mg/kg (milligrams per kilogram) every other week. This is an orphan condition. There are unlikely to be randomized controlled trials comparing the doses. However, the current body of literature suggests that 40 mg/kg (milligrams per kilogram)/week leads to improved outcomes for patients with infantile onset Pompe disease.