Table 1. Gaucher's disease pipeline. Selected compounds on the market or in clinical
development for Type I Gaucher's disease.

Source: BCIQ: BioCentury Online Intelligence

Company

Product

Description

Type of therapy

Status

Genzyme Corp.;
Sanofi (Euronext:SAN; NYSE:SNY)

Cerezyme imiglucerase

Recombinant glucocerebrosidase (GBA; GCase) (rGCase)

Enzyme replacement therapy (ERT)

Marketed

Genzyme; Sanofi

Ceredase alglucerase

Alglucerase enzyme

ERT

Marketed

Protalix BioTherapeutics Inc. (NYSE-M:PLX;
Tel Aviv:PLX); Pfizer Inc. (NYSE:PFE)

Elelyso alfataliglicerase

Plant cell-expressed rGCase

ERT

Marketed

Shire plc

Vpriv velaglucerase alfa

rGCase

ERT

Marketed

Actelion Ltd. (SIX:ATLN); UCB Group (Euronext:UCB)

Zavesca miglustat

Glucosyltransferase inhibitor

Substrate reduction therapy (SRT)

Marketed

Genzyme; Sanofi

Cerdelga eliglustat tartrate

Glucosylceramide synthase inhibitor

SRT

Phase III

Isu Abxis Co. Ltd. (KOSDAQ:086890)

ISU302

Biosimilar Cerezyme

ERT

Phase III

Protalix BioTherapeutics

PRX-112

Plant cell-expressed rGCase (oral)

ERT

Phase I

ExSAR Corporation

EXR-202A

GCase-binding chaperone

Not applicable

Phase I

AThe indication for EXR-202 may include neuronopathic Gaucher's disease.