Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Replagal (agalsidase alfa), Agalsidase Alfa, TAK-675, algalsidase alfa, alpha-Galactosidase, DRX005B, algasidase alfa
Replagal · 7 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.
An adverse event (AE) was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related.Treatment-emergent adverse events (TEAEs) were defined as those events which occurred or worsened in severity after first treatment with Replagal AF until 30 days after the last dose. A serious AE (SAE) was any AE occurred at any dose that resulted in death, life-threatening, hospitalization, prolongation of existing hospitalization, persistent or significant disability or incapacity and congenital anomaly or birth defect.
An IRR (also referred to as infusion-related adverse event \[IRAE\]) was defined as an AE that began either during the infusion or within 12 hours after the start of the infusion and was judged as possibly or probably related to study drug. The IRRs were classified based on the severity as Mild=No limitation of usual activities, Moderate=Some limitation of usual activities, Severe=Inability to carry out usual activities and Life-threatening=Immediate risk of death. The number of participants with infusion-related reactions was reported.
The IgA status was measured using enzyme-linked immunosorbent assay (ELISA). Number of participants who reported positive to IgA was reported.
The IgE status was measured using ELISA. Number of participants who reported positive to IgE was reported.
The IgM status was measured using ELISA. Number of participants who reported positive to IgM was reported.
The ADA status was measured using ELISA and electrochemiluminescent (ECL) immunoassay. Number of participants who reported positive to ADA was reported.
The NAb status was measured using enzyme activity inhibition assay. Number of participants who reported positive to NAb was reported.
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® | EXPERIMENTAL | All eligible patients may receive Replagal produced by the bioreactor process (AF Replagal) on this treatment plan until AF Replagal is commercially available for the patient, the patient's participation is discontinued, or the study is discontinued, whichever comes first. |
| 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, IV, EOW) | EXPERIMENTAL | Screening period of approximately 14 days during which all patients received 1 infusion of 0.2 mg/kg Replagal RB (Week 0) Treatment period of 14 weeks during which all patients received 7 infusions of 0.2 mg/kg Replagal AF |
| Replagal 0.2 mg/kg every other week (EOW) | EXPERIMENTAL | - |
| Agalsidase alfa (Cohort 1) | EXPERIMENTAL | Cohort 1: Patients who completed TKT023. |
| Agalsidase Alfa (Cohort 2) | EXPERIMENTAL | Cohort 2: Treatment-naive patients. |
| Name | Type | Description |
|---|---|---|
| REPLAGAL | BIOLOGICAL | REPLAGAL IV infusion. |
| agalsidase alfa | BIOLOGICAL | Cohort 1: 0.2 mg/kg body weight administered as an intravenous (IV) infusion over 40 minutes every other week (EOW) Cohort 2: 0.2 mg/kg body weight administered as an intravenous (IV) infusion over 40 minutes weekly |
| 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 used to treat Fabry disease, a rare inherited lysosomal storage disorder. It is studied in both adult and pediatric patients, including treatment-naive Chinese participants, children aged 7 years and older, and adults with cardiac involvement. Its clinical program spans four completed trials in Fabry disease.
Replagal is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the sponsor behind the clinical program evaluating Replagal in Fabry disease across multiple countries, including the United States, China, Australia, and several European nations.
Replagal is in Phase 3 development for Fabry disease. Its clinical program includes Phase 3 trials such as NCT04974749, NCT01124643, and NCT00864851, along with a Phase 2 pediatric safety study, NCT01363492. All four trials have been completed.
Replagal has been evaluated in four completed trials: NCT04974749 in treatment-naive Chinese participants, NCT01363492 in children with Fabry disease, NCT01124643 as an extension study in adults, and NCT00864851 comparing dosing regimens on cardiac function in adults. Total enrollment across these studies was 114 participants.
Yes, Replagal is also known as agalsidase alfa. Both names refer to the same therapy developed by Takeda for Fabry disease, so searches using either name point to the same clinical program and the same four completed trials in Fabry disease.
Replagal trials used an active-controlled design, and one study was randomized. The program enrolled 114 participants total across four completed trials, including adults and children aged 7 years and older, with studies conducted in the United States, China, Australia, and multiple European countries.