Familial cold autoinflammatory syndrome 1; FCAS1

Disease ID:1025
Name:Familial cold autoinflammatory syndrome 1; FCAS1
Associated with:1 targets
0 immuno targets immuno relevant
3 immuno ligands
Familial cold urticaria
Database Links
OMIM: 120100
Orphanet: ORPHA47045



Ligand Approved Immuno References Clinical comments
Immuno Disease Comments: Approved therapy for FCAS1.
canakinumab  [ FDA (2009), EMA (2009) (Drugs.com) ]
Clinical Use: Used to treat familial cold autoinflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS) in patients > 4 years of age, and systemic juvenile idiopathic arthritis (SJIA) in patients > 2 years old.
Immuno Disease Comments: An anti-IL-1β therapeutic approved for FCAS1.
rilonacept  [ FDA (2008), EMA (2009) (Drugs.com) ]
Clinical Use: Currently used in the treatment of cryopyrin-associated periodic syndrome (CAPS). Cryopyrin-associated periodic syndrome includes three previously distinct disorders: Familial cold autoinflammatory syndrome; Muckle-Wells syndrome; and CINCA syndrome, that are now considered to represent a disease continuum, all caused by NLRP3 protein mutations.

EMA approval has been withdrawn.
Immuno Disease Comments: Approved drug for FCAS1.