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VX-864 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to VX-864 in the treatment period for 28 days. |
| VX-864 100 mg | EXPERIMENTAL | Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days. |
| VX-864 300 mg | EXPERIMENTAL | Participants received VX-864 300 mg q12h in the treatment period for 28 days. |
| VX-864 500 mg | EXPERIMENTAL | Participants received VX-864 500 mg q12h in the treatment period for 28 days. |
| Name | Type | Description |
|---|---|---|
| VX-864 | DRUG | Tablets for oral administration. |
| Placebo | DRUG | Placebo matched to VX-864 for oral administration. |
Key Inclusion Criteria: * Subjects must have a PiZZ genotype confirmed at screening * Plasma AAT levels indicating severe deficiency at screening Key Exclusion Criteria: * History of a medical condition that could negatively impact the ability to complete the study * Solid organ, or hematological...
VX-864 is an investigational small molecule being developed for the treatment of Alpha1-Antitrypsin Deficiency, a rare genetic condition. It is designed to address the underlying cause of the disease, though the exact mechanism has not been disclosed. The drug is currently in clinical development and is not yet approved for use.
VX-864 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. Vertex is conducting clinical trials to evaluate the safety and efficacy of VX-864 in patients with Alpha1-Antitrypsin Deficiency.
VX-864 is in Phase 2 clinical development. A Phase 2 trial has been completed, but the drug remains investigational and has not received regulatory approval. It is still undergoing clinical evaluation to determine its safety and effectiveness for treating Alpha1-Antitrypsin Deficiency.
VX-864 has been studied in one clinical trial, identified as NCT04474197, titled 'Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype.' This Phase 2, randomized, double-blind, placebo-controlled trial enrolled 44 participants and has been completed. The study was conducted in the United States, Canada, Germany, Ireland, Sweden, and the United Kingdom.
No, VX-864 is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for Alpha1-Antitrypsin Deficiency. The completed Phase 2 trial evaluated its safety and efficacy, but the drug has not yet received marketing approval from regulatory authorities.