Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease. ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease ( 1 ). FDA label
Also known as Plant cell expressed recombinant glucocerebrosidase (prGCD), Plant Cell Expressed Recombinant Human Glucocerebrosidase, prGCD
Taliglucerase alfa · 4 trials · 1 indication
Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Spleen volume measured by MRI
| Arm | Type | Description |
|---|---|---|
| 60 Units/kg | EXPERIMENTAL | - |
| 30 Units/kg | EXPERIMENTAL | - |
| Taliglucerase alfa 30 units/kg | EXPERIMENTAL | Subjects randomized to receive 30 units/kg |
| Taliglucerase alfa 60 units/kg | EXPERIMENTAL | Subjects randomized to 60 units/kg |
| Taliglucerase alfa | EXPERIMENTAL | Open label taliglucerase alfa treatment |
| Naive 30 Units/kg | EXPERIMENTAL | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
| Naive 60 Units/kg | EXPERIMENTAL | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
| Switchover | EXPERIMENTAL | Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348) |
| Name | Type | Description |
|---|---|---|
| Taliglucerase alfa | DRUG | Taliglucerase alfa for infusion every two weeks for 24 months |
Inclusion Criteria: * Successful completion of Protocol PB-06-002 or PB-06-005 * The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent Exclusion Criteria: * Currently taking another investigational drug for any condition. * Presence of neurological signs and symptoms...
Taliglucerase alfa is an investigational enzyme replacement therapy being developed for the treatment of Gaucher Disease, a rare genetic disorder. It is currently in Phase 3 clinical development and has not yet been approved by regulatory authorities.
Taliglucerase alfa is being developed by Pfizer, Inc. (NYSE: PFE). The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Gaucher Disease.
Taliglucerase alfa is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. All four clinical trials for the drug have been completed.
Taliglucerase alfa has been studied in four completed Phase 3 clinical trials. These include NCT00705939, NCT00712348, NCT01411228, and NCT01422187, which evaluated the drug in adult and pediatric patients with Gaucher Disease.
Taliglucerase alfa is a distinct enzyme replacement therapy, though one clinical trial (NCT00712348) involved switching patients from imiglucerase to taliglucerase alfa. The drug is being developed as a potential treatment option for Gaucher Disease.