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AT2221

Phase 3

Pompe Disease (Late-onset) | Small molecule | Metabolic |Amicus Therapeutics, Inc.|Last Updated: Mar 5, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLED
Total Trials1
Total Enrollment119
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04138277A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)PHASE3 COMPLETED 119 — — Dec 18, 2019Dec 31, 2024Mar 5, 202660 United States, Argentina +21
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Study Endpoints
Primary Endpoints
Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug
Entire extension study (mean = 40.5 months on treatment)

Number of subjects with TEAE, TESAE, and TEAE leading to discontinuation during this long-term extension study

Secondary Endpoints
Change From Baseline in 6-Minute Walk Distance (6MWD)
baseline, Week 208
Change From Baseline in Sitting % Predicted Forced Vital Capacity (FVC)
baseline, Week 208
Change From Baseline in Manual Muscle Testing (MMT) Lower Extremity Score
baseline, Week 208
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ATB200/AT2221EXPERIMENTALParticipants received ATB200 (cipaglucosidase alfa) co-administered with AT2221 capsule (miglustat)
Interventions
NameTypeDescription
AT2221DRUGParticipants received ATB200 co-administered with AT2221 (miglustat)
ATB200BIOLOGICALEnzyme Replacement Therapy via intravenous infusion
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites60

Inclusion Criteria: 1\. Subject must have completed Study ATB200-03. Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of cipaglucosidase alfa/miglustat (eg, hospitalization for a car accident, COVID-19 pandemic, or emerge...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBosnia and HerzegovinaCanadaDenmarkFranceGermanyGreeceHungaryItalyJapanNetherlandsNew ZealandPolandSloveniaSouth KoreaSpainSwedenTaiwanUnited Kingdom
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Recent Changes (Last 90 Days)
MEDIUMApr 8, 2026NCT04138277TRIAL_REMOVED: changed
MEDIUMApr 8, 2026NCT04138277TRIAL_REMOVED: changed