Now that the genetic mutation and cause for CAPS has been found, new medications have been discovered, or are being developed that target the main source of inflammation - the over-production and over-secretion of Interleukin 1ß (IL-1ß) by altered cryopyrin inflammasomes.
New drugs can prevent the cellular signaling of Interleukin-1ß (IL-1ß) in CAPS patients. The FDA-approved medications include Kineret (anakinra) that is approved in the US for patients with the NOMID form of CAPS, plus Arcalyst (rilonacept) and Ilaris (canakinumab) that are approved in the US for patients with the FCAS and MWS forms of CAPS. Ilaris was also approved in 2016 by the FDA to also treat HIDS/MKD, TRAPS and FMF, and was approved a few years earlier to treat SJIA. More information about these three treatments are listed below, with links to their prescribing information and approval information for these drugs for other countries outside the US. Please refer to the official drug company websites below to have the most current information about these medications.
This medication is an Interleukin-1ß (IL-1ß) blocking medication. Patients with CAPS have a constant over-production of IL-1ß due to the NLRP3 (CIAS1) genetic mutation. Arcalyst inhibits IL-1ß by attaching and neutralizing IL-1ß in the circulating blood before IL-1ß can bind to the IL-1ß receptors on the surfaces of cells. This can prevent the cells from being triggered to activate increase production of inflammatory mediators. Once IL-1ß is attached to Arcalyst, the IL-1ß is "trapped" and eliminated from the body over time. Arcalyst Drug Information
Arcalyst has proven to be very effective, with a great reduction in inflammation and a decrease in symptoms in FCAS and MWS patients.
Ilaris (canakinumab) is a monoclonal antibody against IL-1ß. The NLRP3 genetic mutation causing CAPS leads to a constant overproduction of IL-1ß by the cryopyrin inflammasome, which left untreated leads to increased inflammation throughout the body.
Ilaris has a strong affinity for human IL-1ß, and can bind specifically to IL-1ß in the body for many weeks. Ilaris is able to block the IL-1ß from interacting with cellular IL-1ß receptors, which prevents IL-1ß triggered activation and the production of increased inflammatory mediators in patients with CAPS. Ilaris does not bind to Il-1 alpha or Il-1 receptor agonist (IL-1 ra) in the body. http://www.centerwatch.com/drug-information/fda-approvals/drug-details.aspx?DrugID=1034
Kineret was also approved to treat patients with all forms of CAPS (FCAS, Muckle-Wells and NOMID/CINCA) in the EU in November 2013, and was approved for the NOMID form of CAPS in the US by the FDA in early 2013.
Kineret (anakinra) is a recombinant, non-glycosylated synthetic form of human Interleukin-1ß receptor antagonist (Il-1 Ra). Natural (endogenous) humanIl-1 Ra is found in small amounts in the body, and regulates the Interleukin 1ß (IL-1ß ). Patients with an over-production of Il-1ß in the body do not have enough of their own Il-1 Ra to control the excessive amount of Il-1ß.
Kineret, a synthetic form of Il-1 RA, acts in the body like the endogenous human Il-1RA to inhibit Il-1ß binding to the Il-1receptor type1 (Il-1 RI), which helps to inhibit the biologic activity of Il-1ß in the body.
Kineret, made by Swedish Orphan Biovitrum (SOBI), was given orphan drug designation (ODD) by the US FDA for the treatment of CAPS in August of 2010. On January 8, 2013 SOBI announced that the US Food and Drug Administration (FDA) has approved Kineret® (anakinra) for the treatment of children and adults with neonatal-onset multisystem inflammatory disease (NOMID). Kineret® is the first and only FDA-approved therapy for NOMID, the most severe form of cryopyrin associated periodic syndromes (CAPS).
How to inject Kineret This is a nice website about Kineret and how it is used for NOMID. Quinn's journey with NOMID This is a wonderful video about one patient's life with NOMID, and how Kineret has helped them.
This is the first approval allowing the use of Kineret in children. Kineret was approved for NOMID under an Orphan Drug designation. A priority review was granted by the FDA based on the product's potential to provide a significant advance in therapy for the NOMID patient population where no adequate therapy exists. Sobi will provide a prefilled syringe with a graduated label to allow flexible dosing in children. Kineret has been approved for the reduction of signs and symptoms of Rheumatoid Arthritis (RA) in adults since 2001. Here is the January 8, 2013 press release from SOBI Press Release Anakinra Drug Information