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Cipaglucosidase Alfa · 3 trials · 2 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.
| 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. |
| 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. |
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 therapy being studied for Glycogen Storage Disease Type II Infantile Onset and Pompe Disease (Late-onset). It is a monoclonal antibody developed by Amicus Therapeutics, Inc. (NASDAQ: FOLD) and is currently in Phase 3 clinical development for these rare disease indications.
Cipaglucosidase Alfa is a monoclonal antibody designed to treat Pompe disease by providing enzyme replacement therapy. It targets the underlying enzyme deficiency in patients with Glycogen Storage Disease Type II, helping to address the accumulation of glycogen in tissues.
Cipaglucosidase Alfa is developed by Amicus Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol FOLD. The company is conducting Phase 3 clinical trials to evaluate the safety and efficacy of this investigational therapy for Pompe disease.
Cipaglucosidase Alfa is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials are ongoing to assess its safety and efficacy in patients with late-onset Pompe disease and infantile-onset Glycogen Storage Disease Type II.
Cipaglucosidase Alfa is being evaluated in three Phase 3 trials. NCT03729362 is a completed study in adults with late-onset Pompe disease. NCT03911505 is an active but not recruiting study in pediatric patients with late-onset Pompe disease. NCT04808505 is currently recruiting for infantile-onset Glycogen Storage Disease Type II.
Cipaglucosidase Alfa is the same as ATB200. Clinical trial titles refer to the combination of ATB200/AT2221, where ATB200 is cipaglucosidase alfa and AT2221 is miglustat, a chaperone therapy. This combination is being studied in Phase 3 trials for Pompe disease.