Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Etavopivat A · 1 trial · 2 indications
Measured as number of events.
Measured as number of adverse reactions.
| Arm | Type | Description |
|---|---|---|
| Participants greater than or equal to (≥) 12 years old with sickle cell disease | EXPERIMENTAL | Participants will receive an oral dose of Etavopivat A or C. |
| Participants ≥ 12 years old with sickle cell disease transfusion dependent | EXPERIMENTAL | Participants will receive an oral dose of Etavopivat A or C. |
| Participants ≥ 12 years old with transfusion-dependent thalassaemia | EXPERIMENTAL | Participants will receive an oral dose of Etavopivat A or C. |
| Participants ≥ 12 years old with non-transfusion dependent thalassaemia | EXPERIMENTAL | Participants will receive an oral dose of Etavopivat A or C. |
| Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease | EXPERIMENTAL | Participants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants \< 12 years of age will receive an oral dose of Etavopivat B. |
| Name | Type | Description |
|---|---|---|
| Etavopivat A | DRUG | Participants will receive an oral dose of Etavopivat A. |
| Etavopivat B | DRUG | Participants will receive an oral dose of Etavopivat B. |
| Etavopivat C | DRUG | Participants will receive an oral dose of Etavopivat C. |
Inclusion Criteria: * Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study. * Participant must have derived clinical benefit from treatment with etavopi...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Etavopivat A is an investigational small molecule being developed for the treatment of Sickle Cell Disease. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities. The drug is also being studied in people with thalassaemia as part of a long-term treatment research study.
Etavopivat A targets and activates the PKLR gene product, pyruvate kinase. As a PKLR activator, it is designed to modulate red blood cell metabolism. This mechanism is being investigated as a potential therapeutic approach for sickle cell disease, a hematologic condition.
Etavopivat A is being developed by Novo Nordisk A/S, a pharmaceutical company listed on the stock exchange under the ticker NVO. The company is conducting clinical research to evaluate the drug's safety and efficacy in patients with sickle cell disease.
Etavopivat A is currently in Phase 3 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 3 trial is recruiting participants to study long-term treatment with the drug.
Etavopivat A is being studied in a Phase 3 clinical trial registered as NCT06609226. This recruiting study is examining long-term treatment with Etavopivat in people with sickle cell disease or thalassaemia, with an estimated enrollment of 480 participants across multiple countries.
Etavopivat A is a form of the drug Etavopivat. The name Etavopivat A is used in clinical development contexts, and the drug is also referred to simply as Etavopivat. Both names refer to the same investigational PKLR activator being developed by Novo Nordisk.