Where can Fabrazyme (agalsidase β) be obtained for a Fabry disease patient?

Where to Obtain Fabrazyme (Agalsidase Beta)

Fabrazyme is manufactured by Genzyme Corporation (Cambridge, Massachusetts) and is obtained through specialty pharmacy distribution networks after FDA approval and insurance authorization. 1

Regulatory Approval and Availability

• Fabrazyme received FDA approval and is commercially available in the United States as a prescription medication for Fabry disease 1
• The European Agency for the Evaluation of Medicinal Products (EMEA) has also approved Fabrazyme in the European Union 2
• Fabrazyme is supplied as a sterile, lyophilized powder in two vial sizes: 35 mg and 5 mg vials for reconstitution with Sterile Water for Injection 1

Practical Acquisition Process

Prescription Requirements

• A physician experienced in managing Fabry disease or inherited metabolic diseases must prescribe and supervise treatment 2
• The prescription requires documentation of confirmed Fabry disease diagnosis through enzyme activity testing (males) or DNA-based molecular testing (females) 2

Distribution Channels

• Fabrazyme is distributed through specialty pharmacies that handle high-cost biologic medications requiring special storage and handling
• The medication requires cold chain management as it is a temperature-sensitive biologic product 1
• Coordination with Genzyme Corporation's patient support programs facilitates access and addresses supply issues 2

Insurance and Access Considerations

Medical Necessity Documentation

• Current renal function (serum creatinine, eGFR, 24-hour urine protein or spot urine protein-to-creatinine ratio) must be documented 3
• Baseline organ involvement assessment including cardiac evaluation (echocardiography for left ventricular mass), pain quantification, and plasma GL-3 or lyso-Gb3 levels strengthens authorization 3
• Every male Fabry patient should be offered enzyme replacement therapy regardless of chronic kidney disease stage 2

Treatment Initiation Criteria

• Symptomatic females with chronic acroparesthesias resistant to conventional therapy, persistent proteinuria (>300 mg/24 hours), GFR below 80 mL/minute/1.73 m², or clinically significant cardiac involvement qualify for treatment 2
• Asymptomatic females require annual monitoring, while symptomatic patients need evaluation every six months 2

Critical Supply Management

Dosing and Administration

• The approved dosage is 1 mg/kg administered intravenously every 2 weeks 1, 4
• Each infusion requires advance ordering to ensure adequate supply, as this is a specialty medication with limited distribution 1

Common Pitfalls to Avoid

• Do not delay treatment initiation waiting for advanced organ damage, as benefits on estimated GFR and serum creatinine are more pronounced when therapy begins at less advanced stages of renal dysfunction 2, 3
• Approximately 57-59% of patients develop infusion-associated reactions; premedication protocols should be established before the first infusion 3
• Stopping enzyme replacement therapy in symptomatic patients risks irreversible organ damage, particularly renal fibrosis and cardiac complications 3

Monitoring During Treatment

• Plasma GL-3 levels should normalize (≤7.03 µg/mL) and remain normal with continued treatment 1
• Follow-up evaluations should occur every 6 months for symptomatic patients to assess treatment effectiveness through renal function, cardiac parameters, and pain reduction 2, 3
• Higher anti-agalsidase beta antibody titers are associated with less robust GL-3 clearance and should be monitored 3