Fragile X Syndrome

Rare Disease·MeSH: Fragile X Syndrome

Prevalence

30.0 / 100K

11 drugs in development·4 companies·5 clinical trials

Drug Pipeline by Phase

Phase 3(5)

Drug	Company
ANAVEX®2-73	ANAVEX LIFE SCIENCES CORP. (AVXL)
Transdermal cannabidiol therapies (Zynerba)	Harmony Biosciences Holdings, Inc. (HRMY)
ZYN-002	Harmony Biosciences Holdings, Inc. (HRMY)
ZYN002	Harmony Biosciences Holdings, Inc. (HRMY)
zyn002 - transdermal gel	Zynerba Pharmaceuticals, Inc. (ZYNE)

Phase 2(5)

Drug	Company
High dose of MRM-3379	Mirum Pharmaceuticals, Inc. (MIRM)
Low dose of MRM-3379	Mirum Pharmaceuticals, Inc. (MIRM)
Low dose of MRM-3379 Open-Label	Mirum Pharmaceuticals, Inc. (MIRM)
Middle Dose of MRM-3379	Mirum Pharmaceuticals, Inc. (MIRM)
MRM-3379	Mirum Pharmaceuticals, Inc. (MIRM)

Phase 2/Phase 3(1)

Drug	Company
zyn002 - cannabidiol transdermal gel	Zynerba Pharmaceuticals, Inc. (ZYNE)

BioSniper tracks 11 drugs in development for Fragile X Syndrome across 4 companies.

There are 5 clinical trials targeting Fragile X Syndrome tracked by BioSniper.

The estimated prevalence of Fragile X Syndrome is approximately 30.0 per 100,000 population.

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