Sickle cell disease (SCD) is a rare blood condition. It causes sickling in the red blood cells which results in the blockage of blood vessels and capillaries. The sickling process also causes hemolytic anemia which can damage the body’s organs and ultimately result in early death. The disease presents in childhood and patients deal with various physical and psychosocial disabilities as a result of the pain caused by blocked blood flow.
But, positive results just recently announced from a Phase 1/2 study could mean a new therapeutic option for patients with SCD may be on the way.
Global Blood Therapeutics conducted a Phase 1/2 study to examine the effect of voxelotor in individuals with SCD. Data from this study were published in Blood.
Voxelotor has already received Breakthrough Therapy, Orphan Drug, Fast Track, and Rare Pediatric Disease designation by the FDA for SCD. It is an oral therapy that SCD patients would take once each day. It works by blocking polymerization in the blood and therefore, sickling.
The drug has shown it can reduce hemolysis, sickling, and anemia. It was shown to be tolerable, safe, and effective for SCD patients in the study.
The Phase 1/2 trial included 38 participants who received doses of voxelotor at 500mg, 700mg, or 1000mg once each day for 28 days. 16 participants received doses of 700mg, 900mg, or placebo for 90 days. 4 of the participants from the latter group also participated in an extension study. These individuals received a 900mg dose of the drug each day for 6 months. Overall, participants who received the drug for 90 days or more showed a rise in hemoglobin and a reduction in hemolysis as well as a reduction in sickled cells. As anemia and hemolysis are the largest causes of organ damage and other series complications associated with the disease, these results for voxelotor are very promising.
Global Blood Therapeutics hopes to submit a New Drug Application to the FDA for voxelotor this year and already has a meeting planned with the FDA to discuss the filing. They are also planning a transcranial study later this year.
Researchers believe this drug could ultimately become the new standard treatment for SCD.
Global Blood therapeutics recently completed their HOPE study of voxelotor which was a Phase 3 trial. This trial included individuals as young as age 12. The company also has an ongoing study examining the effects of the drug in pediatric patients. This trial is Phase 2a and it’s called HOPE-KIDS 1. It includes patients age 4-17.
Hopefully we will see this potentially life-altering drug become available for patients with sickle cell disease in the near future.
You can read more about the latest Phase 1/2 study and the progress on voxelotor here.