Pompe Disease

Rare Disease·MeSH: Glycogen Storage Disease Type II

Prevalence

0.5 / 100K

Incidence

0.3 / 100K/yr

21 drugs in development·9 companies·12 clinical trials

Drug Pipeline by Phase

Approved(3)

Drug	Company
pombiliti	AMICUS THERAPEUTICS, INC. (FOLD)
Pombiliti + Opfolda	AMICUS THERAPEUTICS, INC. (FOLD)
Pombiliti™ + Opfolda™	AMICUS THERAPEUTICS, INC. (FOLD)

Phase 3(7)

Drug	Company
Alglucosidase Alfa	AMICUS THERAPEUTICS, INC. (FOLD)
Alglucosidase alfa or Avalglucosidase alfa	AMICUS THERAPEUTICS, INC. (FOLD)
AT2221	AMICUS THERAPEUTICS, INC. (FOLD)
ATB200	AMICUS THERAPEUTICS, INC. (FOLD)
Cipaglucosidase Alfa	AMICUS THERAPEUTICS, INC. (FOLD)
Duvoglustat	AMICUS THERAPEUTICS, INC. (FOLD)
Miglustat	AMICUS THERAPEUTICS, INC. (FOLD)

Phase 2(2)

Drug	Company
rhGAA	AMICUS THERAPEUTICS, INC. (FOLD)
S-606001	Valneva SE (VALN)

Phase 1(3)

Drug	Company
DNL952	Denali Therapeutics Inc. (DNLI)
mze001	Maze Therapeutics, Inc. (MAZE)
spk-3006	Spark Therapeutics, Inc. (SPARK)

Preclinical(4)

Drug	Company
atx-04	AVENUE THERAPEUTICS, INC. (ATXI)
Pompe program candidate	Dyne Therapeutics, Inc. (DYN)
sl65-based candidates	LogicBio Therapeutics, Inc. (LOGC)
spk-gaa	Spark Therapeutics, Inc. (SPARK)

Unknown(2)

Drug	Company
avalglucosidase alfa-ngpt	Sanofi (SNY)
avalglucosidase alfa-ngpt iv	Sanofi (SNY)

BioSniper tracks 21 drugs in development for Pompe Disease across 9 companies.

There are 12 clinical trials targeting Pompe Disease tracked by BioSniper.

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

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