Table 3 Historic and available therapies for Gaucher disease
Medication name | Therapy type | Advantages | Disadvantages |
|---|---|---|---|
Alglucerase (Ceredase, Genzyme corporation) | Enzyme replacement | Earliest therapy with demonstrated improvements in organ involvement, biomarkers, bone pain. Satisfactory safety profile | No longer available Derived from human placenta Intravenous route Cost Does not cross blood-brain barrier Allergic reactions |
Imiglucerase (Cerezyme, Genzyme corporation) | Enzyme replacement | Replaced alglucerase with comparable therapeutic response Satisfactory safety profile | Intravenous route Cost Does not cross blood-brain barrier Allergic reactions |
Velaglucerase alfa (VPRIV, Shire Human Genetics Therapies) | Enzyme replacement | Fewer allergic reactions Comparable therapeutic response with imiglucerase | Intravenous route Cost Does not cross blood-brain barrier |
Taliglucerase alfa (Elelyso, Pfizer Inc.) | Enzyme replacement | Easier manufacturing, lower cost | Intravenous route Does not cross blood-brain barrier Less therapeutic response data |
Miglustat (Zavesca, Actelion) | Substrate reduction | Oral route Potential to cross blood-brain barrier | Failed to achieve neurological treatment response High prevalence of side effects Cost |
Eliglustat (Genzyme corporation) | Substrate reduction | Oral route Early clinical evidence of treatment response | Drug-drug interactions CYP2D6 and CYP3A metabolism considerations Cardiotoxicity Does not cross blood-brain barrier Cost |