Recent developments, utilization, and spending trends for pompe disease therapies

Area of Science:

• Biochemistry
• Pharmacology
• Genetics

Background:

• Pompe disease is a rare inherited metabolic myopathy caused by acid alpha-glucosidase (GAA) deficiency.
• Infantile Pompe disease has a low survival rate (25.7% to age 1) with no prior therapies before 2006.
• This condition affects lysosomal cells, leading to progressive muscle weakness.

Purpose of the Study:

• To review recent Pompe disease therapies, including two FDA-approved biologic drugs.
• To analyze drug utilization and spending trends for Pompe disease treatments in the US Medicaid program.
• To compare the indications, efficacy, and safety profiles of newly approved Pompe disease therapies.

Main Methods:

• Reviewed two recently approved Pompe disease therapies, comparing indications, efficacy, and safety.
• Conducted a retrospective analysis of the national Medicaid pharmacy claims database.
• Calculated quarterly prescriptions and reimbursement amounts from Q2 2006 to Q2 2011.

Main Results:

• Myozyme (alglucosidase alfa) and Lumizyme (alglucosidase alfa) were FDA-approved in 2006 and 2010, respectively.
• Both therapies improve survival but carry boxed warnings for allergic reactions; Lumizyme has restricted distribution.
• Medicaid spending for Myozyme and Lumizyme increased significantly, with average per-prescription costs around $10,000 and $20,000, respectively.

Conclusions:

• Medicaid beneficiaries show rising utilization and spending for Myozyme and Lumizyme.
• The per-prescription average price for both Pompe disease therapies remained relatively steady.
• Ongoing research is exploring new therapeutic options for Pompe disease.