Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
GZ402666 · 1 trial · 3 indications
| Arm | Type | Description |
|---|---|---|
| GZ402666 (neoGAA) Group 1 - 5 mg | EXPERIMENTAL | Intravenous infusion of 5mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks |
| GZ402666 (neoGAA) Group 1 - 10 mg | EXPERIMENTAL | Intravenous infusion of 10mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. |
| GZ402666 (neoGAA) Group 1 - 20 mg | EXPERIMENTAL | Intravenous infusion of 20mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. |
| GZ402666 (neoGAA) Group 2 - 5 mg | EXPERIMENTAL | Intravenous infusion of 5mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. |
| GZ402666 (neoGAA) Group 2 - 10 mg | EXPERIMENTAL | Intravenous infusion of 10mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. |
| GZ402666 (neoGAA) Group 2 - 20 mg | EXPERIMENTAL | Intravenous infusion of 20mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. |
| Name | Type | Description |
|---|---|---|
| GZ402666 | DRUG | Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous |
Inclusion criteria : For both Group 1 and Group 2: * Male or female patients with confirmed acid α-glucosidase (GAA) enzyme deficiency from any tissue source and/or confirmed GAA gene mutation and without known cardiac hypertrophy. * Patient willing and able to provide signed informed consent * Pa...
GZ402666 is an investigational small molecule being developed by Sanofi for the treatment of Pompe Disease, also known as Glycogen Storage Disease Type II (GSD II) or Acid Maltase Deficiency. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
GZ402666 is being developed by Sanofi, a multinational pharmaceutical company traded on the stock exchange under the ticker symbol SNY. The drug is an investigational small molecule intended for the treatment of Pompe Disease, a rare genetic disorder.
GZ402666 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The drug is being studied for the treatment of Pompe Disease, also known as Glycogen Storage Disease Type II.
GZ402666 has been studied in one completed Phase 1 clinical trial with the identifier NCT01898364. This trial evaluated the safety and efficacy of repeat dosing in late onset Pompe Disease patients and enrolled 24 participants across multiple countries including the United States, Belgium, Denmark, France, Germany, Netherlands, and the United Kingdom.
GZ402666 is associated with the trial NCT01898364, which evaluates repeat neoGAA dosing in late onset Pompe Disease patients. The trial title refers to neoGAA, suggesting that GZ402666 may be the same compound as neoGAA, though this is not explicitly confirmed in the available data.