Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Sickle Cell Disease

Treatment Oxbryta (voxelotor)

The insurer denied coverage for Oxbryta (voxelotor)

The denial is overturned

This is an adult male with a history of sickle cell disease and iron deficiency. It was noted his hemoglobin (Hgb) was 8.2. He was unable to tolerate Hydrea. The treating provider is requesting the plan's approval for Oxbryta (voxelotor).

According to OXBRYTA HIGHLIGHTS OF PRESCRIBING INFORMATION, "OXBRYTA is a hemoglobin S [inherited from parents] polymerization inhibitor indicated for the treatment of sickle cell disease in adults and pediatric patients of preteen age and older."

Per Howard, J. et al., " Voxelotor 1500 mg [milligrams] resulted in rapid and durable improvements in hemoglobin concentrations maintained over 72 weeks and has potential to address the substantial morbidity associated with hemolytic anemia in sickle cell disease."

Lastly, Vichinski, E. et al., writes, "A total of 274 participants were randomly assigned in a 1:1:1 ratio to receive a once-daily oral dose of 1500 mg of voxelotor, 900 mg of voxelotor, or placebo. Most participants had sickle cell anemia (homozygous hemoglobin S or hemoglobin), and approximately two thirds were receiving hydroxyurea at baseline. In the intention-to-treat analysis, a significantly higher percentage of participants had a hemoglobin response in the 1500-mg voxelotor group (51% [percent]; 95% confidence interval [CI], 41 to 61) than in the placebo group (7%; 95% CI, 1 to 12). Anemia worsened between baseline and week 24 in fewer participants in each voxelotor dose group than in those receiving placebo. At week 24, the 1500-mg voxelotor group had significantly greater reductions from baseline in the indirect bilirubin level and percentage of reticulocytes than the placebo group. The percentage of participants with an adverse event that occurred or worsened during the treatment period was similar across the trial groups. Adverse events of at least grade 3 occurred in 26% of the participants in the 1500-mg voxelotor group, 23% in the 900-mg voxelotor group, and 26% in the placebo group. Most adverse events were not related to the trial drug or placebo, as determined by the investigators. In this phase 3 randomized, placebo-controlled trial involving participants with sickle cell disease, voxelotor significantly increased hemoglobin levels and reduced markers of hemolysis. These findings are consistent with inhibition of hemoglobin S (HbS) polymerization and indicate a disease-modifying potential. (Funded by Global Blood Therapeutics; HOPE ClinicalTrials.gov number, NCT03036813. opens in new tab)."

Oxbryta is Food and Drug Administration (FDA)-approved to be used to improve hemoglobin in patients with sickle cell disease who are anemic. This agent was studied with or without Hydrea and in patients who were Hydrea-intolerant. 

This is a patient with symptomatic anemia in the setting of sickle cell disease. The use of Oxbryta was medically necessary treatment for the patient's condition, as supported by current medical literature and any applicable diagnostic/treatment guidelines.

The health care plan did not act reasonably and with sound medical judgment or in the best interest of the patient

The insurer's denial of coverage for Oxbryta (voxelotor) to treat sickle cell disease is overturned. Medical necessity is substantiated.