Drug2nd line3 diseases

Rilonacept

IL-1 decoy receptor (IL-1 Trap)

Response rate
Significant improvement vs placebo
Onset
Days–weeks
Route
SC 160mg load, then 80mg weekly
Line
2nd
IgM effect
Improved
Evidence level
amber
Trial phase
Phase 4

Used across diseases

DiseaseResponse rateLineEvidence
Cryopyrin-Associated Periodic SyndromesSignificant improvement vs placebo2ndamber
Familial Mediterranean FeverModerate3rdamber
Schnitzler SyndromeLimited dataAlternativedefault

Evidence summary

Dimeric fusion protein acting as IL-1 'trap' neutralizing IL-1α and IL-1β. FDA-approved for FCAS and MWS (2008). Two sequential placebo-controlled studies demonstrated efficacy. Less widely used than anakinra/canakinumab due to limited long-term data and availability.

Approved indications

Conditions for which Rilonacept has regulatory approval (not specific to rare diseases covered here):

FCASMWSRecurrent Pericarditis

Drug identifiers

DrugBankDB06372
ATC CodeL04AC04
Open TargetsCHEMBL1201561

Sources (1)

DetailsHoffman HM et al. (2008) Efficacy and safety of rilonacept in CAPS: results from two sequential placebo-controlled studiesDOI