Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Alglucosidase alfa (GZ419829), Myozyme, Lumizyme
Alglucosidase alfa · 9 trials · 9 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 |
|---|---|---|
| 1 | EXPERIMENTAL | - |
| 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. |
| Name | Type | Description |
|---|---|---|
| Myozyme | BIOLOGICAL | 20 mg/kg qow |
| alglucosidase alfa | BIOLOGICAL | IV infusion of 20mg/kg; qow for 78 weeks. |
| Placebo | DRUG | Placebo Comparator; qow for 78 weeks. |
Inclusion Criteria: * male or female aged greater than or equal to 18 years * patient's legally authorized guardian(s) must provide signed, informed consent prior to initiation of study; patient's signature required if patient understands informed consent * patient must have a documented deficit in...
Alglucosidase alfa is used for Glycogen Storage Disease Type II and late-onset Pompe Disease. It is an investigational therapy being developed by Sanofi for these rare lysosomal storage disorders, which are characterized by acid maltase deficiency and glycogen accumulation.
Alglucosidase alfa targets the GAA enzyme, which is deficient in patients with Pompe disease. By addressing this molecular target, the therapy aims to replace the missing enzyme activity in patients with Glycogen Storage Disease Type II and late-onset Pompe Disease.
Sanofi (ticker: SNY) is developing Alglucosidase alfa. The company is conducting clinical research on this therapy for the treatment of Glycogen Storage Disease Type II and late-onset Pompe Disease.
Alglucosidase alfa is in Phase 2 clinical development. It remains investigational and has not been approved by regulatory authorities. The therapy is being studied for Glycogen Storage Disease Type II and late-onset Pompe Disease.
Alglucosidase alfa has been studied in two completed clinical trials: NCT00158600, a Phase 3 placebo-controlled study in 90 patients with late-onset Pompe Disease, and NCT00051935, a Phase 2 study in 2 siblings with Glycogen Storage Disease Type II.
Yes, Alglucosidase alfa is also known as GZ419829. Researchers and clinicians may refer to the therapy by either name when discussing its development for Glycogen Storage Disease Type II and late-onset Pompe Disease.