Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Alglucosidase alfa (GZ419829)
alglucosidase alfa · 2 trials · 6 indications
Overall safety summary of patients experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment, i.e., alglucosidase alfa or placebo.
Mean distance walked gives an indication of functional endurance. The greater the distance, the greater the endurance. Mean values of distance walked in a six-minute walk test are offered for baseline, week 78 (or last available observation), and the mean change from baseline (at week 78 or last available post-baseline observation).
Forced vital capacity is a standard pulmonary function test used to quantify respiratory muscle weakness. Forced vital capacity (FVC) is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%.
Area under the plasma concentration versus time curve from time zero (pre-dose) to 16 hours after the end of infusion. Blood sample time points were 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Time to maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
| Arm | Type | Description |
|---|---|---|
| alglucosidase alfa | ACTIVE_COMPARATOR | Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks. |
| Placebo | PLACEBO_COMPARATOR | Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks. |
| 1 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| alglucosidase alfa | BIOLOGICAL | IV infusion of 20mg/kg; qow for 78 weeks. |
| Placebo | DRUG | Placebo Comparator; qow for 78 weeks. |
Inclusion Criteria: * Patient must provide signed, informed consent prior to performing any study-related procedures. * Patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean of the testi...
Alglucosidase alfa is used for Glycogen Storage Disease Type II, also known as Pompe Disease, specifically in the late-onset form. It is an investigational therapy being developed by Sanofi for this rare disease.
Alglucosidase alfa targets the underlying enzyme deficiency in Pompe Disease. It is a form of recombinant human acid alpha-glucosidase (rhGAA) designed to replace the missing enzyme that leads to glycogen accumulation in cells.
Sanofi (ticker: SNY) is developing Alglucosidase alfa. The drug is being studied for the treatment of Glycogen Storage Disease Type II and late-onset Pompe Disease.
Alglucosidase alfa is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. One Phase 2 trial has been completed, along with a Phase 3 trial.
Alglucosidase alfa has been studied in two completed trials. NCT00051935 was a Phase 2 study in siblings with Glycogen Storage Disease Type II, and NCT00158600 was a Phase 3 placebo-controlled study in patients with late-onset Pompe Disease.
Yes, Alglucosidase alfa is also known as GZ419829. This alternative name may be used in some clinical or research contexts to refer to the same drug.