FDA Approves Voxelotor for Pediatric Sickle Cell Patients

The United States Food and Drug Administration (FDA) has approved voxelotor (Oxbryta) for patients with sickle cell disease (SCD) in pediatric patients ages 4-12.

The additional New Drug Application (sNDA) was awarded to Global Blood Therapeutics and built on previous approval for patients with sickle cell disease aged 12 years and older.

The treatment is the first approved treatment that directly targets the polymerization of sickle hemoglobin, the main cause of the sickle formation and destruction of red blood cells in SCD.

“For decades, the sickle cell disease community has been severely underreported and there are limited treatment options for younger patients with their entire lives ahead of them. Complications of SCD that can cause irreversible organ damage are known to begin in the first few years of life, and therefore, earlier intervention is critical,” Ted W. Love, MD, president and chief executive officer of GBT, said in a statement.

“The FDA approval of Oxbryta for children as young as 4 years old – and in a pediatric-friendly dosage form – is an important step forward in the treatment of this devastating, lifelong condition. GBT is proud to lead the development of new drugs to address the shortcomings of care for SCD patients.”

The FDA also approved a separate New Drug Application (NDA) for voxelotor tablets for oral suspension, a dispersible, once-daily tablet dosage form suitable for patients aged 4-12 years and for elderly patients who have difficulty swallowing whole tablets. .

Recently, researchers observed that voxelotor increased hemoglobin (Hb) by ≥1 g/dL, with study data indicating statistically significant reductions in transfusions, vaso-occlusive crises (VOCs), and VOC-related all-cause hospitalizations following voxelotor use.

The study was presented at the 2021 American Society of Hematology (ASH) Annual Meeting & Exposition.

Among the subgroup of patients with pre- and post-voxelotor Hb measurements (n = 63), mean Hb (95% confidence interval) was 7.9 (7.5 – 8.2) g/dL at baseline and final Hb 8.9 (8.4 – 9.4 ) g/dL after voxelator initiation.

Data show that most patients (38 of 63, 60.3%) achieved an Hb rise of >1 g/dL at some point during the follow-up period.

In patients who received ≥1 transfusion (n = 357) in the year before starting voxelotor, the mean transfusion rate decreased by 43% from 3.2 (2.8 – 3.7) to 1.8 (1.4 – 2 .3) PPY (P < .001).

Furthermore, in 40 patients receiving chronic transfusions (≥8) the mean transfusion rate decreased by 34%, from 9.8 (8.3 – 11.4) to 6.5 (4.4 – 8.5) PPY (P = .007).

Among those who had ≥1 VOC in the 3 months prior to starting the voxelator, the mean annualized VOC percentage decreased by 22%, from 10.9 (10.4 – 11.5) to 8.5 (7 .8 – 9.3) (P < 0.001). In addition, the mean number of VOC-related hospitalizations decreased by 32%, from 7.3 (6.9 - 7.7) to 5.0 (4.4 - 5.6) (P < .001).

Subsequently, in 636 patients who had been hospitalized 3 months prior to initiating voxelotor, the mean all-cause hospitalization rate was reduced by 36%, from 7.5 (7.1 – 7.9) to 4.8 (4.3 – 5.3) after treatment (P < 0.001).

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