FDA approves voxelotor for sickle cell disease

FDA approves voxelotor for sickle cell disease

On November 25th, 2019, the Food and Drug Administration (FDA) granted accelerated approval to voxelotor (Oxbryta, Global Blood Therapeutics) for adults and paediatric patients 12 years of age older with sickle cell disease (SCD). SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of haemoglobin (HbS). Mutated HbS has a propensity to polymerize, or bind together, forming long, rigid rods within a red blood cell (RBC). The polymer rods deform RBCs to assume a sickled shape and to become inflexible, which causes hemolytic anemia (low hemoglobin due to RBC destruction).

Efficacy of voxelotor was evaluated in 274 patients with sickle cell disease in a randomized, double-blind, placebo-controlled, multicentre trial. Patients were randomized to voxelotor 1500 mg (N=90), 900 mg (N=92), or placebo (N=92). The median age was 24 years (range 12, 64). Patients were enrolled if their baseline hemoglobin (Hb) ≥5.5 to ≤10.5 g/dL. Randomization was stratified by whether the patient was already receiving hydroxyurea, by geographic region, and by age.

In Europe, on 18th November 2016, orphan designation (EU/3/16/1769) was granted by the European Commission to SynteractHCR Deutschland GmbH, for voxelotor. Currently, the only medicine authorised in the EU to treat sickle cell disease was hydroxycarbamide. The main treatment for sickle cell disease was blood transfusion. This was usually combined with ‘iron chelators’ (medicines used to treat iron overload, high iron levels in the body caused by repeated blood transfusions), which are necessary in patients with long-term anaemias such as sickle cell disease. Moreover, the European Medicines Agency (EMA) has included voxelotor in its Priority Medicines (PRIME) program, a scheme launched by EMA to enhance support for the development of medicines that target an unmet medical .

Currently, just two clinical trials (Phase I and Phase II) were performed and terminated in Europe on patients aged 18 to 60. If on one side in USA, the Global Blood Therapeutics is currently evaluating volexotor in the ongoing Phase 2a HOPE-KIDS 1 Study, an open-label, single- and multiple-dose study in pediatric patients (age 4 to 17) with SCD, on the other side in Europe the situation is far to define a solution in the paediatric field.

December 12th, 2019|