Adakveo (crizanlizumab-tmca) — Medical Mutual

Sickle cell disease

Initial criteria

Patient is at least age ≥ 16 years; AND
Therapy will not be used in conjunction with voxelotor (Oxbryta) or L-glutamine (Endari); AND
Patient has not received prior treatment with gene therapy (i.e., lovotibeglogene autotemcel, exagamglogene autotemcel); OR Patient failed to respond or lost response to treatment with prior gene therapy (i.e., lovotibeglogene autotemcel, exagamglogene autotemcel); AND
Patient has a confirmed diagnosis of sickle-cell disease, of any genotype (e.g., HbSS, HbSC, HbS/beta0-thalassemia, HbS/beta+-thalassemia, and others) as determined by either identification of significant quantities of HbS with or without an additional abnormal β-globin chain variant by hemoglobin assay OR identification of biallelic HBB pathogenic variants where at least one allele is the p.Glu6Val pathogenic variant on molecular genetic testing; AND
Patient had an insufficient response to a minimum 3-month trial of hydroxyurea (unless contraindicated or intolerant); AND
Patient experienced one or more vaso-occlusive crises (VOC) in the previous year despite adherence to hydroxyurea therapy

Patient continues to meet the universal and other indication-specific relevant criteria; AND
Absence of unacceptable toxicity from the drug (e.g., severe infusion-related reactions such as pain, nausea, vomiting, diarrhea, fatigue, dizziness, pruritus, pyrexia); AND
Evidence of disease response compared to pretreatment baseline as demonstrated by decreased frequency of vaso-occlusive crises necessitating treatment, reduction in number or duration of hospitalizations, and/or reduction in severity of VOC

6 months initial, 12 months renewal