Pompe Disease (Late-onset)

Rare Diseases
11
Pipeline Programs
5
Companies
9
Clinical Trials
1 recruiting
2
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
3
1
0
4
3
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
1100%
+ 9 programs with unclassified modality

On Market (2)

Approved therapies currently available

Sanofi
LUMIZYMEApproved
alglucosidase alfa
Sanofi
Hydrolytic Lysosomal Glycogen-specific Enzyme [EPC]iv (infusion)2010
Sanofi
MYOZYMEApproved
alglucosidase alfa
Sanofi
iv (infusion)2006

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Sanofi
SanofiPARIS, France
4 programs
1
1
1
Alglucosidase AlfaPhase 41 trial
MyozymePhase 31 trial
MyozymePhase 21 trial
Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and N/A1 trial
Active Trials
NCT00731081Completed8Est. May 2009
NCT00250939Completed5Est. Nov 2006
NCT00268944Completed5Est. Jun 2007
+1 more trials
Amicus Therapeutics
Amicus TherapeuticsPA - Philadelphia
3 programs
3
AT2221Phase 31 trial
Cipaglucosidase AlfaPhase 31 trial
Cipaglucosidase AlfaPhase 31 trial
Active Trials
NCT04138277CompletedEst. Dec 2024
NCT03911505Active Not RecruitingEst. Jun 2026
NCT03729362CompletedEst. Jan 2021
AskBio
AskBioNC - Durham
1 program
1
AB-1009Phase 1/21 trial
Active Trials
NCT07282847Recruiting12Est. Sep 2032
Bayer
BayerLEVERKUSEN, Germany
1 program
1
AB-1009Phase 1/2
Astellas
AstellasChina - Shenyang
1 program
1
zocaglusagene nuzaparvovecPhase 1/2Gene Therapy1 trial
Active Trials
NCT04174105Active Not Recruiting11Est. Feb 2035

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
SanofiAlglucosidase Alfa
Amicus TherapeuticsCipaglucosidase Alfa
Amicus TherapeuticsAT2221
Amicus TherapeuticsCipaglucosidase Alfa
SanofiMyozyme
SanofiMyozyme
AskBioAB-1009
Astellaszocaglusagene nuzaparvovec
SanofiStudy About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and

Clinical Trials (9)

Total enrollment: 57 patients across 9 trials

NCT01288027SanofiAlglucosidase Alfa

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Start: Jun 2011Est. completion: Dec 201316 patients
Phase 4Completed
NCT03911505Amicus TherapeuticsCipaglucosidase Alfa

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Start: Feb 2020Est. completion: Jun 2026
Phase 3Active Not Recruiting

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Start: Dec 2019Est. completion: Dec 2024
Phase 3Completed
NCT03729362Amicus TherapeuticsCipaglucosidase Alfa

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Start: Dec 2018Est. completion: Jan 2021
Phase 3Completed

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

Start: Dec 2005Est. completion: Jun 20075 patients
Phase 3Completed

A Study of rhGAA in Patients With Late-Onset Pompe Disease

Start: Feb 2005Est. completion: Nov 20065 patients
Phase 2Completed

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

Start: Feb 2026Est. completion: Sep 203212 patients
Phase 1/2Recruiting
NCT04174105Astellaszocaglusagene nuzaparvovec

Gene Transfer Study in Patients With Late Onset Pompe Disease

Start: Oct 2020Est. completion: Feb 203511 patients
Phase 1/2Active Not Recruiting
NCT00731081SanofiStudy About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and

Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

Start: Mar 2007Est. completion: May 20098 patients
N/ACompleted

Related Jobs in Rare Diseases

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

4 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 57 patients
5 companies competing in this space