Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as alglucosidase alfa, Alglucosidase alfa GZ419829, Recombinant human acid glucosidase, Lumizyme
Myozyme · 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...
Myozyme is used to treat Pompe disease, also known as glycogen storage disease type II. The listed indications cover infantile-onset Pompe disease and late-onset Pompe disease. It is developed by Sanofi and studied in patients with these forms of the condition, including those receiving respiratory support in one trial.
Myozyme is developed by Sanofi, which trades under the ticker SNY. The clinical trial records for Myozyme reference Genzyme-sponsored enzyme replacement therapy studies, and Genzyme is part of Sanofi. Sanofi is the company associated with the development and sponsorship of the Myozyme trials listed.
Myozyme has been studied in Phase 2 and Phase 3 clinical trials. The listed studies include Phase 2 extension studies and a Phase 3 safety and effectiveness study. All four listed trials are completed, and none are currently active. The program covers infantile-onset and late-onset Pompe disease.
Myozyme has been evaluated in completed trials including NCT00765414, an extension study in a single late-onset Pompe disease patient, NCT00763932, an extension study in Pompe disease patients previously enrolled in Genzyme enzyme replacement therapy studies, NCT00268944, a Phase 3 study in advanced late-onset Pompe disease patients receiving respiratory support, and NCT00250939, a Phase 2 study in late-onset Pompe disease.
The listed Myozyme trials enrolled a total of 52 patients across four completed studies. Enrollment was small, ranging from 1 patient in the single-patient extension study NCT00765414 to 7 patients in the extension study NCT00763932. The other two studies each enrolled 5 patients.
Yes, Myozyme is studied under the name rhGAA, which stands for recombinant human acid alpha-glucosidase, in several of its clinical trials. Trial titles for NCT00268944 and NCT00250939 refer to rhGAA in patients with late-onset Pompe disease, and these studies describe the same glycogen storage disease type II population treated with Myozyme.