50
Management
Table 13B. Food and Drug Administration (FDA)-Approved
Precision Agents With Applications in IEI
Therapy Use in IEI
JAK inhibitors JAK/STAT GOF disorders, interferonopathies
Elapegademase
ADA deficiency SCID
a
Anakinra
NOMID
a
, DIRA
a
Canakinumab
FCAS
a
, MWS
a
, NOMID
a
, FMF, TRAPS, HIDS/MKD
Rilonacept
FCAS
a
/MWS
a
Abatacept CTLA-4 haploinsufficiency
Plerixafor WHIM syndrome
Mavorixafor
WHIM syndrome
a
Cyanocobalamin TCN2 deficiency
Hydroxocobalamin
Folinic acid SLC46A1 deficiency
MTHFD1 deficiency
IFN-γ 1-b
CGD
a
and immune disorders of interferon signaling
Leniolisib
APDS
a
a
FDA-approved indication.
