Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as ATB200
Cipaglucosidase Alfa · 4 trials · 3 indications
The efficacy of cipaglucosidase alfa/miglustat co-administration on ambulatory function was measured by the 6MWT. The 6MWD, measured in meters, is the distance walked on the 6MWT. A greater distance indicated greater endurance. An increase from baseline indicated improvement.
Number of subjects with TEAE, TESAE, and AE leading to discontinuation during the 2 year treatment period and extension (Stage 3 and 4 combined)
Plasma GAA levels (Cmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
Plasma GAA levels (Tmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
Plasma GAA levels (AUC) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
| Arm | Type | Description |
|---|---|---|
| Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects | EXPERIMENTAL | Pediatric IOPD subjects 6 months to \<18 years experiencing clinical decline |
| Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects | EXPERIMENTAL | Pediatric IOPD subjects \<6 months |
| Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) | EXPERIMENTAL | Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule |
| Cipaglucosidase Alfa/Miglustat | EXPERIMENTAL | Participants received cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W). |
| Alglucosidase Alfa/Placebo | ACTIVE_COMPARATOR | Participants received alglucosidase alfa co-administered with placebo Q2W. |
| ATB200 | EXPERIMENTAL | Sequential single ascending doses of intravenously infused ATB200 for 3 dosing periods |
| ATB200 + AT2221 | EXPERIMENTAL | ATB200 co-administered with AT2221 (Miglustat) |
| Name | Type | Description |
|---|---|---|
| Cipaglucosidase alfa | BIOLOGICAL | Sterile lyophilized powder intravenous (IV) infusion |
| Miglustat | DRUG | 65 mg oral capsules |
| Alglucosidase Alfa | BIOLOGICAL | Participants received an IV infusion dose over a 4-hour duration Q2W. |
| Placebo | DRUG | Miglustat matching placebo was administered orally 1 hour prior to alglucosidase alfa infusion Q2W. |
| ATB200 | DRUG | - |
| AT2221 | DRUG | - |
Inclusion Criteria: Cohort 1: 1. Male or female subjects who are aged 6 months to \< 18 years on Day 1 2. Subject must have documentation of IOPD genotype 3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis 4. Subject must have received ERT for at least 6 months immediate...
Cipaglucosidase alfa is an investigational enzyme replacement therapy being developed for Pompe disease, also known as glycogen storage disease type II. It is being studied in two forms of the condition: infantile-onset Pompe disease in patients under 18 years of age and late-onset Pompe disease. It is not yet approved.
Cipaglucosidase alfa targets acid alpha-glucosidase (GAA), the enzyme that is deficient in Pompe disease. By supplying a recombinant form of this enzyme, the therapy is designed to address the underlying enzyme deficiency that leads to glycogen accumulation in muscle tissue.
Cipaglucosidase alfa is being developed by Amicus Therapeutics, Inc., which trades on the Nasdaq under the ticker FOLD. The company is running the clinical development program for the asset across infantile-onset and late-onset Pompe disease populations.
Cipaglucosidase alfa is in Phase 3 clinical development. It is an investigational therapy and has not been approved by the FDA. The program includes one completed Phase 3 trial, one active trial that is no longer recruiting, and one recruiting Phase 3 study.
Cipaglucosidase alfa has been studied in three Phase 3 trials. NCT03729362 compared it with alglucosidase alfa in adults with late-onset Pompe disease and is completed. NCT03911505 is an open-label pediatric late-onset study that is active but not recruiting. NCT04808505 is recruiting infants and children with infantile-onset disease.
Across the three Phase 3 studies, 182 participants have been enrolled. NCT03729362 enrolled 125 adults with late-onset Pompe disease, NCT04808505 is set to enroll 36 patients with infantile-onset disease, and NCT03911505 enrolled 21 pediatric patients with late-onset disease.