Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Replagal · 4 trials · 1 indication
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this investigational product (IP) or medicinal product. Serious AE was any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality/birth defect, and was an important medical event. A TEAE was defined as any event emerging at or after the initiation of treatment with an IP or any existing event that worsened in either intensity or frequency following exposure to the IP until the end of the safety follow-up period.
Left ventricular mass (LVM) was measured through echocardiography.
Reflects development of Anti-Agalsidase antibodies post baseline
| Arm | Type | Description |
|---|---|---|
| REPLAGAL | EXPERIMENTAL | Participants received REPLAGAL 0.2 milligrams per kilogram (mg/kg) body weight, intravenous (IV) infusion, every other week (EOW) from Day 1 (Week 0) up to Week 52. |
| Replagal 0.2 mg/kg EOW | EXPERIMENTAL | Intravenous, 0.2mg/kg EOW |
| Replagal 0.2 mg/kg, IV, every other week | ACTIVE_COMPARATOR | Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every other week for 52 weeks. |
| Replagal 0.2 mg/kg, IV, weekly | ACTIVE_COMPARATOR | Patients randomized to receive Replagal 0.2 mg/kg via intravenous infusion every week for 52 weeks. |
| Replagal 0.4 mg/kg, IV, weekly | ACTIVE_COMPARATOR | Patients randomized to receive Replagal 0.4 mg/kg via intravenous infusion every week for 52 weeks. |
| Replagal 0.2 mg/kg every other week (EOW) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| REPLAGAL | BIOLOGICAL | REPLAGAL IV infusion. |
| Replagal (agalsidase alfa) | BIOLOGICAL | 0.2 mg/kg administered over 40 minutes every other week (EOW) |
Inclusion Criteria: * Participant and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the participant. Fo...
Replagal is an investigational treatment for Fabry Disease, a rare genetic disorder. It is being developed as a therapy to address the underlying condition in affected patients. The drug has been studied in clinical trials involving adults and children with Fabry Disease to evaluate its safety and clinical outcomes.
Replagal is developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company traded on the New York Stock Exchange under the ticker TAK. Takeda is responsible for the clinical development and regulatory activities for Replagal in Fabry Disease.
Replagal is in Phase 3 clinical development for Fabry Disease. It has completed four clinical trials, including Phase 3 studies in adults and a Phase 2 study in children. The drug remains investigational and is not yet approved for commercial use.
Replagal has completed four clinical trials, including NCT00864851, a Phase 3 study of dosing regimens on cardiac function in adults; NCT01124643, an extension study in adults; NCT01363492, a Phase 2 safety study in children; and NCT04974749, a Phase 3 study in treatment-naive Chinese participants. All trials are completed.
Replagal is the brand name for agalsidase alfa, an enzyme replacement therapy for Fabry Disease. The drug is developed by Takeda Pharmaceutical Company Limited and has been studied in multiple clinical trials to evaluate its safety and efficacy in patients with Fabry Disease.