Fabry Disease

Rare Disease·MeSH: Fabry Disease

Prevalence

1.0 / 100K

Incidence

0.2 / 100K/yr

16 drugs in development·9 companies·22 clinical trials

Drug Pipeline by Phase

Approved(3)

Drug	Company
elfabrio	Protalix BioTherapeutics, Inc. (PLX)
Galafold	AMICUS THERAPEUTICS, INC. (FOLD)
Galafold®	AMICUS THERAPEUTICS, INC. (FOLD)

Phase 3(6)

Drug	Company
ERT	AMICUS THERAPEUTICS, INC. (FOLD)
migalastat	Sanofi (SNY)
migalastat	AMICUS THERAPEUTICS, INC. (FOLD)
migalastat	Sanofi (SNYNF)
migalastat HCl	AMICUS THERAPEUTICS, INC. (FOLD)
venglustat	Sanofi (SNY)

Phase 1(1)

Drug	Company
4D-310	4D Molecular Therapeutics, Inc. (FDMT)

Preclinical(1)

Drug	Company
sig-007	Sigilon Therapeutics, Inc. (SGTX)

Phase 1/Phase 2(4)

Drug	Company
amt-191	uniQure N.V. (QURE)
isaralgagene civaparvovec	SANGAMO THERAPEUTICS, INC (SGMO)
Isaralgagene civaparvovec (ST-920)	SANGAMO THERAPEUTICS, INC (SGMO)
ST-920	SANGAMO THERAPEUTICS, INC (SGMO)

Phase 4(1)

Drug	Company
replagal	SHIRE PLC (SHPGF)

BioSniper tracks 16 drugs in development for Fabry Disease across 9 companies.

There are 22 clinical trials targeting Fabry Disease tracked by BioSniper.

The estimated prevalence of Fabry Disease is approximately 1.0 per 100,000 population.

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