Sickle cell disease (SCD) is among the world’s most common serious inherited diseases and that worsens over time. Treatments are available that can prevent complications and lengthen the lives of those suffering from this disorder.
The U.S. Food and Drug Administration granted accelerated approval to Voxelotor to treat Sickle Cell Disease in adults and children 12 years or older.
Voxelotor is an oral, once-daily therapy for the treatment of SCD. It is a sickle haemoglobin (HbS) polymerization inhibitor and it works by a novel mechanism of action increasing the affinity of haemoglobin for oxygen. This prevents haemoglobin polymerization and the resultant sickling and destruction of the red blood cells.
The approval was based on the results of a clinical trial with 274 patients with SCD; moreover, nonclinical studies demonstrated Voxelotor efficacy in improving red blood cell deformability and the ability to flow. However, further clinical trials are required to verify and describe Voxelotor clinical benefit.
The European Medicines Agency (EMA) has included Voxelotor in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated Voxelotor as an orphan medicinal product for the treatment of patients with SCD.
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