Revcovi — Medical Mutual

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Initial criteria

Patient does not have severe thrombocytopenia (i.e., platelet count <50,000/microL); AND
Patient has adenosine deaminase severe combined immunodeficiency (ADA-SCID) disease as determined by one of the following:
Deficient ADA catalytic activity (<1% of normal) in hemolysates (in untransfused individuals) or in extracts of other cells (e.g., blood mononuclear cells, fibroblasts); OR
Detection of biallelic pathogenic mutations in the ADA gene by molecular genetic testing; AND
Patient has elevated deoxyadenosine triphosphate (dATP) or total deoxyadenosine nucleotides (dAXP) in red blood cells; AND
Patient is not a candidate for or has failed definitive therapy with bone marrow transplantation (BMT); OR
Patient is a candidate for definitive therapy with BMT and elapegademase will be used as bridge therapy; AND
Patient has baseline values for trough plasma ADA activity, red blood cell dATP, trough red blood cell dAXP, and/or total lymphocyte counts

Patient continues to meet the universal and other indication-specific relevant criteria such as concomitant therapy requirements, performance status, etc.; AND
Absence of unacceptable toxicity from the drug (examples include injection site bleeding in patients with thrombocytopenia, severe thrombocytopenia, delay in improvement of immune function, etc.); AND
Patient has demonstrated a beneficial response to therapy compared to pretreatment baseline in one or more of the following:
Increase in plasma ADA activity (target trough level ≥ 15 mmol/hr/L)
Decrease in red blood cell dATP level (target ≤ 0.005 to 0.015 mmol/L)
Improvement in immune function with diminished frequency/complications of infection as evidenced in improvement in the ability to produce antibodies
Decrease in red blood cell dAXP level (target trough level ≤ 0.02 mmol/L)
Increase in total lymphocyte counts

12 months