Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Etavopivat Tablets Low dose, Etavopivat Low dose, Etavopivat tablets, Etavopivat B, Etavopivat C, FT-4202
Etavopivat · 12 trials · 5 indications
Measured as Count of events.
Measured as number of events.
Measured as number of adverse reactions.
Hemoglobin response rate at Week 24 (increase of \> 1 g/dL \[\> 10 g/L\] from baseline) during the blinded treatment period
Annualized vaso-occlusive crisis rate during the 52-week blinded treatment period based on adjudicated vaso-occlusive crisis review
Change in cerebral blood flow (CBF) assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Change in CMRO2 assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history
Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history
Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline)
Measured as count of events.
Measured as milliseconds.
Measured as picograms per milliliter (pg/mL).
Measured as hours\*picograms per milliliter (h\*pg/mL).
Measured as pg/mL.
Measured as h\*pg/mL.
Measured as pg/mL.
Measured as h\*pg/mL.
Measured as nanograms per milliliter (ng/mL).
Measured as hours\*nanograms per milliliter(h\*ng/mL).
Measured as ng/mL.
Measured as h\*ng/mL.
Measured in hour\*nanogram/millilitre (h\*ng/mL).
Measured in nanograms/millilitre (ng/mL).
Measured in(hour\*nanogram/millilitre)/kilograms ((h\*ng/mL)/kg).
Measured in (nanograms/millilitre )/kilogram ((ng/mL)/kg).
Measured in hour\*nanogram/millilitre (h\*ng/mL).
Measured in hour (h).
Measured in hour (h).
Measured in Litre/hour (L/h).
Measure in litre (L).
Measured as hours nanograms per milliliter (h\*ng/mL).
Measured as nanograms per milliliter (ng/mL).
Measured in hours\*nanogram per milliter (h\*ng/mL).
Measured in nanogram per milliter (ng/mL).
| Arm | Type | Description |
|---|---|---|
| Etavopivat | EXPERIMENTAL | Participants will be randomised to receive oral dose of Etavopivat. |
| Placebo | PLACEBO_COMPARATOR | Participants will be randomised to receive oral dose of placebo. |
| 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. |
| Double blind etavopivat Low Dose | EXPERIMENTAL | Double blind etavopivat Low Dose |
| Double blind etavopivat High Dose | EXPERIMENTAL | Double blind etavopivat High Dose |
| Double blind placebo | EXPERIMENTAL | Double blind placebo |
| Open label etavopivat | EXPERIMENTAL | Open label etavopivat |
| Etavopivat with HU | EXPERIMENTAL | Participants will receive Etavopivat 400 mg QD orally in combination with HU. The dose of HU (mg/kg) will be stable (no more than a 20% change in dosing except for weight-based changes) during the study, in the opinion of the Investigator. |
| Cohort 1: Etavopivat (12 to less than [<] 18 years) | EXPERIMENTAL | Participants aged 12 to less than 18 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group. |
| Cohort 2: Etavopivat (6 to <12 years) | EXPERIMENTAL | Participants aged 6 to less than 12 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group. |
| Cohort 3: Etavopivat (2 to <6 years) | EXPERIMENTAL | Participants aged 2 to less than 6 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in younger children. |
| Cohort 4: Etavopivat (6 months to <2 years) | EXPERIMENTAL | Participants aged 6 months to less than 2 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in infants. |
| Etavopivat 400 mg daily - SCD with transfusions | EXPERIMENTAL | Patients with sickle cell disease on chronic red blood cell transfusions |
| Etavopivat 400 mg daily - Thalassemia with transfusions | EXPERIMENTAL | Patients with thalassemia on chronic red blood cell transfusions |
| Etavopivat 400 mg daily - Thalassemia | EXPERIMENTAL | Patients with thalassemia not on chronic red blood cell transfusions |
| Part A- Etavopivat | EXPERIMENTAL | Participants will receive a single dose of etavopivat. |
| Part A- Placebo | PLACEBO_COMPARATOR | Participants will receive a single dose of placebo. |
| Part B- Etavopivat | EXPERIMENTAL | Participants will receive a single dose of etavopivat. |
| Part B- Moxifloxacin | OTHER | Participants will receive a single dose of moxifloxacin. |
| Part B- Placebo | PLACEBO_COMPARATOR | Participants will receive a single dose of placebo. |
| Period 1: digoxin+pitavastatin+metformin | EXPERIMENTAL | Participants will receive a single dose of oral digoxin, pitavastatin and metformin. |
| Period 1: midazolam+rosuvastatin | EXPERIMENTAL | Participants will receive a single dose of oral midazolam and rosuvastatin. |
| Period 2: etavopivat+midazolam | EXPERIMENTAL | Participants will receive a daily dose of oral etavopivat and single dose of oral midazolam. |
| Period 2: etavopivat+midazolam+rosuvastatin | EXPERIMENTAL | Participants will receive a daily dose of oral etavopivat, single dose of oral midazolam and rosuvastatin. |
| Period 2: etavopivat+digoxin+pitavastatin+metformin | EXPERIMENTAL | Participants will receive a daily dose of oral etavopivat, single dose of oral digoxin, pitavastatin and metformin. |
| Sequence 1: Etavopivat: fed-fasted | EXPERIMENTAL | Participants will receive a single dose of Etavopivat in fed condition in period 1 and a single dose of Etavopivat in fasted condition in period 2. |
| Sequence 2: Etavopivat: fasted-fed | EXPERIMENTAL | Participants will receive a single dose of Etavopivat in fasted condition in period 1 and a single dose of Etavopivat in fed condition in period 2. |
| Mild hepatic impairment: Etavopivat dose 2 | EXPERIMENTAL | Participants will receive a single dose of oral etavopivat. |
| Moderate hepatic impairment: Etavopivat dose 2 | EXPERIMENTAL | Participants will receive a single dose of oral etavopivat. |
| Healthy matched controls: Etavopivat dose 2 | EXPERIMENTAL | Participants will receive a single dose of oral etavopivat. |
| Severe hepatic impairment: Etavopivat dose 1 | EXPERIMENTAL | Participants will receive a single dose of oral etavopivat. |
| Healthy matched controls: Etavopivat dose 1 | EXPERIMENTAL | Participants will receive a single dose of oral etavopivat. |
| Name | Type | Description |
|---|---|---|
| Etavopivat | DRUG | Etavopivat will be administered orally. |
| Placebo | DRUG | Placebo matching Etavopivat will be administered orally. |
| 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. |
| Etavopivat Tablets Low dose | DRUG | 200 mg once daily |
| Etavopivat Tablets High dose | DRUG | 400 mg once daily |
| Placebo Tablets | DRUG | Placebo once daily |
| Etavopivat Tablets | DRUG | Selected dose once daily |
| Hydroxyurea | DRUG | Participants will receive a stable dose of HU. |
| Moxifloxacin | DRUG | Moxifloxacin will be administered orally. |
| Digoxin | DRUG | Participants will receive a single dose of digoxin orally. |
| Pitavastatin | DRUG | Participants will receive a single dose of pitavastatin orally. |
| Metformin | DRUG | Participants will receive a single dose of metformin orally. |
| Midazolam | DRUG | Participants will receive a single dose of midazolam orally. |
| Rosuvastatin | DRUG | Participants will receive a single dose of rosuvastatin orally. |
Inclusion Criteria: * Male or female. * Age 12 years or above at the time of signing the informed consent. * Confirmed diagnosis of sickle cell disease: Documentation of sickle cell disease (SCD) genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of lab...
Top 12 of 13 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 6 | PHASE3 | Etavopivat |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab, OTQ923, ITU512 |
| Agios Pharmaceuticals, Inc. | AGIO | 3 | PHASE3 | Mitapivat, Tebapivat |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, rilzabrutinib |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE3 | L-Citrulline |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470 |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
Etavopivat is an investigational oral medicine being studied for sickle cell disease and thalassemia, two inherited blood disorders. It is also being evaluated in liver diseases. It is not approved for any use and remains in clinical development.
Etavopivat is a small molecule activator of PKLR, the gene encoding pyruvate kinase in red blood cells. Activating this enzyme target is intended to improve red blood cell energy metabolism and survival, which is the rationale for studying it in sickle cell disease and thalassemia.
Etavopivat is developed by Novo Nordisk A/S, which trades under the ticker NVO. Novo Nordisk is the sponsor responsible for the clinical development program across sickle cell disease, thalassemia, and liver diseases.
Etavopivat is in Phase 1 development overall, with nine trials recorded and five currently active. Four trials have been completed. It is investigational and has not been approved by the FDA or any other regulator.
Etavopivat is being studied in sickle cell disease and thalassemia trials including NCT06612268, a Phase 3 study in sickle cell disease, and NCT06609226, a Phase 3 long-term treatment study. Earlier completed Phase 1 studies include NCT07023029 and NCT06813924 in healthy participants.
Yes. Etavopivat tablets, etavopivat low dose, etavopivat tablets low dose, and the labels etavopivat A, etavopivat B, and etavopivat C all refer to the same investigational drug, etavopivat, developed by Novo Nordisk.