Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication OXBRYTA is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older. This indication is approved under accelerated approval based on increase in hemoglobin (Hb) [see Clinical Studies (14) ] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). FDA label
Also known as Voxelotor, Oxbryta
GBT440 · 10 trials · 7 indications
Number of participants with increase in Hb \>1 g/dL from Baseline to Week 24
AUCt was calculated using the linear/log trapezoidal rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
Maximum observed plasma concentration
Time at which maximum concentration was observed
Area under the concentration-time curve
Terminal elimination half-life
| Arm | Type | Description |
|---|---|---|
| Dose 1 | ACTIVE_COMPARATOR | voxelotor |
| Dose 2 | ACTIVE_COMPARATOR | voxelotor |
| Placebo | PLACEBO_COMPARATOR | Placebo |
| Part A | EXPERIMENTAL | To evaluate the effect of multiple doses of voxelotor on the plasma pharmacokinetics (PK) of a single dose of bupropion, repaglinide, flurbiprofen, omeprazole, and midazolam |
| Part B | EXPERIMENTAL | To evaluate the effect of multiple doses of voxelotor on the plasma PK of a single dose of metformin, furosemide, and rosuvastatin |
| GBT440 Dose 1:Mild hepatic impairment | EXPERIMENTAL | Child Pugh A |
| GBT440 Dose 1:Moderate hep. impairment | EXPERIMENTAL | Child Pugh B |
| GBT440 Dose 1:Severe hepatic impairment | EXPERIMENTAL | Child Pugh C |
| GBT440 Dose 1:Normal hepatic function | EXPERIMENTAL | Healthy subjects |
| Name | Type | Description |
|---|---|---|
| voxelotor | DRUG | - |
| Placebo | OTHER | - |
| GBT440 | DRUG | Capsules which contain GBT440 drug substance in Swedish orange |
| Bupropion | DRUG | Drug drug interaction |
| Repaglinide | DRUG | Drug drug interaction |
| Flurbiprofen | DRUG | Drug drug interaction |
| Omeprazole | DRUG | Drug drug interaction |
| Midazolam | DRUG | Drug drug interaction |
| Metformin | DRUG | Drug drug interaction |
| Furosemide | DRUG | Drug drug interaction |
| Rosuvastatin | DRUG | Drug drug interaction |
Inclusion Criteria: 1. Male or female study participants with sickle cell disease 2. Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months. 3. Age 12 to 65 years 4. Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening 5. For participants taking hydroxyurea (H...
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 |
GBT440 is an investigational small molecule developed by Pfizer, Inc. (ticker PFE) for hematology conditions. It has been studied in sickle cell disease, anemia, hepatic impairment, renal impairment, and idiopathic pulmonary fibrosis. GBT440 is also known as voxelotor. It is not approved and remains in clinical development.
GBT440 has been studied in sickle cell disease, including a Phase 2 extension study for patients who previously participated in the GBT440-001 study. Research has also covered anemia, hepatic impairment, renal impairment, and idiopathic pulmonary fibrosis. GBT440 is investigational and has not been approved for any indication.
Pfizer, Inc. develops GBT440. The company trades under the ticker PFE. Pfizer is the sponsor behind the clinical program for GBT440, which is also known as voxelotor.
GBT440 is in Phase 1 development overall, with six completed trials. One completed trial, the extension study in sickle cell disease, was Phase 2. GBT440 is investigational and has not been approved by the FDA for any indication.
GBT440 has been studied in six completed trials. These include NCT05981365, a Phase 1 drug interaction study in sickle cell disease; NCT03161015, a Phase 1 single-dose pharmacokinetic study in renal impairment; NCT03114540, a Phase 1 pharmacokinetic and safety study in hepatic impairment; and NCT03041909, a Phase 2 extension study in sickle cell disease.
Yes, GBT440 is the same as voxelotor. Voxelotor is the alternative name for GBT440, the investigational small molecule developed by Pfizer, Inc. (ticker PFE) for sickle cell disease and related hematology conditions. Searches for either name refer to the same drug candidate.