Gaucher Disease

Rare Disease·MeSH: Gaucher Disease

Prevalence

0.5 / 100K

9 drugs in development·7 companies·9 clinical trials

Drug Pipeline by Phase

Approved(3)

Drug	Company
elelyso	Protalix BioTherapeutics, Inc. (PLX)
miglustat	ANI PHARMACEUTICALS INC (ANIP)
vpriv	SHIRE PLC (SHPGF)

Phase 3(2)

Drug	Company
velaglucerase alfa	TAKEDA PHARMACEUTICAL CO LTD (TKPHF)
venglustat	Sanofi (SNY)

Phase 1(1)

Drug	Company
venglustat	Sanofi (SNY)

Phase 1/Phase 2(2)

Drug	Company
ly3884961	Prevail Therapeutics Inc. (PRVL)
pr001	Prevail Therapeutics Inc. (PRVL)

Phase 4(1)

Drug	Company
N-acetylcysteine	ENANTA PHARMACEUTICALS INC (ENTA)

BioSniper tracks 9 drugs in development for Gaucher Disease across 7 companies.

There are 9 clinical trials targeting Gaucher Disease tracked by BioSniper.

The estimated prevalence of Gaucher Disease is approximately 0.5 per 100,000 population.

Full trial data, competitive analysis, and AI-powered pipeline tracking.