OxbrytaTreatment of haemolytic anaemia in sickle cell disease
New molecular entity
Global Blood Therapeutics
Global Blood Therapeutics
Development and Regulatory status
Jul 22Approved in UK "for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adult and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea)" with orphan status [19,20].
Feb 22Approved in EU .
Jan 22Global Blood Therapeutics announces that the MHRA has awarded a positive scientific opinion–under Early Access to Medicines Scheme (EAMS) for voxelotor once-daily tablet for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years-of-age and older. Voxelotor will be offered as monotherapy or in combination with hydroxycarbamide to patients meeting the eligibility criteria while the MHRA continue to review the marketing authorisation application .
Dec 21Recommended for EU approval by CHMP “for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide.” Oxbryta will be available as 500 mg film-coated tablets .
Jun 21MHRA grants Promising Innovative Medicine (PIM) designation to voxelotor for the treatment of sickle cell disease.
Jan 21EMA begins review of MAA for voxelotor for treatment of haemolytic anaemia in sickle cell disease .
Jun 20Manufacturer announces intention to submit a MAA to the EMA for treatment of hemolytic anemia in SCD patients ages 12 years and older "by mid 2021" .
Apr 20NICE received regulatory timing information from the company in Jun 19, and decided that holding a scoping exercise would not be appropriate at that time. It has yet to be rescheduled .
Nov 19Oxbryta launched in the US alongside GBT Source™, a comprehensive support program for patients and physicians [10,11].
Nov 19GBT expect Oxbryta to be available through their speciality pharmacy partner network within two weeks. List price will be $10,417 per month, or $125,000 per year [8,9]
Nov 19Approved in US for treatment of sickle cell disease in adults and pediatric patients 12 years of age and older .
Sep 19The US FDA has accepted for filing the company’s New Drug Application seeking accelerated approval for voxelotor .
Jan 18Jan 18: PIII trial (NCT03036813) extended beyond the US, including to UK, Netherlands and France .
Sep 17FDA grants rare paediatric disease designation for treatment of sickle cell disease .
Jun 17EMA grants Priority Medicines (PRIME) status to GBT 440. Also has orphan drug status in US and fast track status .
Nov 16Granted orphan drug status in EU for treatment of sickle cell anaemia .
An orally available small molecule using its SHAPE platform technology that modifies the shape of sickle haemoglobin (HbS) to a form that prefers the oxygenated state.
Sickle cell disease is thought to be the most common severe genetic disease in the UK and France, with 10,000-15,000 people affected .
Treatment of haemolytic anaemia in sickle cell disease
Trial or other data
Dec 21Positive results from real-world retrospective analysis of voxelotor in 3,128 SCD pts in the USA demonstrated a statistically significant improvement in Hb levels and significant reductions in transfusions, vaso-occlusive crises (VOC) and hospitalisations. Amoung pts with >1 recent transfusion from a health claims database, there was a 52% mean reduction in the number of transfusions, 23% reduction in the number of VOCs and decrease of 34% in hospitalisations. 
Apr 21PIII GBT_HOPE RCT (NCT03036813, n=274) found treatment resulted in improvements in haemoglobin concentrations vs. placebo (mean change baseline to week 72: 1.0, 0.5 and 0.0 g/dL in 1500mg, 900 mg and placebo groups, respectively) .
Jun 19PIII RCT (NCT03036813; n=274) reports 1500mg/day oral voxelotor significantly increased haemoglobin levels and reduced markers of haemolysis compared to placebo; haemoglobin response 51% vs. 7%, respectively (P<0.001) .
Jun 18Positive top-line data from Part A of the PIII HOPE trial of Voxelotor in Sickle Cell Disease. The primary endpoint (the proportion of pts with >1g/dL increase in Hb vs. baseline), was achieved with 58% of pts on voxelotor 1500mg and 38% of pts on voxelotor 900mg exceeding 1g/dL increase in Hb vs. 9% on placebo (p<0.0001 for 1500mg vs. placebo and p<0.0021 for 900mg vs. placebo) at Week 12. There were numerically fewer VOC episodes in both voxelotor groups vs. placebo. Voxelotor was generally safe and well tolerated with similar safety profiles between the two doses. There was no evidence of tissue hypoxia at either dose. Part A of the study aimed to select the optimal dose for Part B and define the outcome measures.
Jan 17PIII HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) starts (NCT03036813). The primary efficacy endpoint is proportion of patients who achieve a >1 g/dL increase in hemoglobin at 24 weeks of treatment vs. baseline. 400 patients (>12 years of age) with sickle cell disease who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year will be enrolled in the US. The trial will be conducted in two parts; Part A will compare two dose levels (900mg and 1500mg) of GBT 440 against placebo in up to 150 patients. Part B will randomise 250 patients to the optimal dose identified in Part A or placebo. Collection of the primary outcome is due to complete Jun 19 .