Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Cipaglucosidase alfa
ATB200 · 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...
ATB200 is an investigational small molecule being studied for the treatment of Pompe Disease, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is administered intravenously and is being evaluated for its safety, tolerability, and pharmacokinetic profile.
ATB200 is a small molecule designed to treat Pompe Disease, a lysosomal storage disorder. While the specific molecular target is not disclosed, the drug is being studied as a potential therapy for this condition. It is administered intravenously and is currently in Phase 1 clinical trials.
ATB200 is being developed by Amicus Therapeutics, Inc., a biopharmaceutical company listed on NASDAQ under the ticker symbol FOLD. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Pompe Disease.
ATB200 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The Phase 1 trial has been completed, and the drug is still in the early stages of clinical testing for Pompe Disease.
ATB200 has been studied in one clinical trial, identified as NCT02675465. This was a first-in-human study to evaluate the safety, tolerability, and pharmacokinetics of intravenous ATB200 alone and when co-administered with oral AT2221. The trial enrolled 29 participants with Pompe Disease and has been completed.
No, ATB200 and AT2221 are different drugs. ATB200 is the investigational drug being studied for Pompe Disease, while AT2221 is an oral medication that was co-administered with ATB200 in the clinical trial. The study evaluated the effects of ATB200 alone and in combination with AT2221.