Current Treatments

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.

Arcalyst was approved in late 2020 by the FDA for the treatment of DIRA, and anakinra was also approved to treat DIRA by the FDA. More info with each medication below.

Healthier Ever After Our original book and program for patients that need injectable medications.

Arcalyst (Rilonacept)

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.

Arcalyst (Rilonacept), made by Regeneron, was the first FDA-approved drug in the USA to treat two forms of CAPS: Muckle-Wells Syndrome (MWS) and Familial Cold Autoinflammatory Syndrome (FCAS)

The FDA approved Arcalyst in 2008 for patients with MWS or FCAS that are 12 years and older. The drug is given as a weekly subcutaneous injection.

Arcalyst was approved in late 2020 by the FDA for the treatment of DIRA. Current Arcalyst Drug information:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/125249s047lbl.pdf

Ilaris (Canakinumab)

Ilaris (canakinumab) is a monoclonal antibody against IL-1ß. This medication has been approved in the US to treat not only some forms of CAPS, but also SJIA, TRAPS, HIDS/MKD and FMF (often for patients that their FMF symptoms are not fully controlled on colchicine) 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

Ilaris Drug information:ilaris.com

Current FDA prescribing info for Ilaris:https://www.novartis.us/sites/www.novartis.us/files/ilaris.pdf

This drug was approved by the FDA in the US in June of 2009 for patients with the Muckle Wells (MWS) and Familial Cold Autoinflammatory Syndrome (FCAS) forms of CAPS.It was later approved for use for SJIA, FMF,MKD/HIDS,and TRAPS.

Ilaris was approved for use in all forms of CAPS (including NOMID/CINCA) in the European Union and Switzerland in 2009, and for FMF. MKD.HIDS,TRAPS and SJIA later.

In 2010, Ilaris was approved in Canada for patients with CAPS, and indicated approval for patients with FCAS and MWS. It has also been approved for use for FMF, TRAPS, MKD/HIDS and SJIA. For use in the NOMID form of CAPS, the current Canadian informations states "ILARIS® may also be used in Neonatal-Onset Multisystem Inflammatory Disease (NOMID)/ Chronic Infantile Neurological, Cutaneous, Articular Syndrome (CINCA). Clinical data have not confirmed improvement in CNS symptoms in patients with this phenotype." https://www.novartis.ca/sites/www.novartis.ca/files/ilaris_scrip_e.pdf

Kineret (Anakinra)-now FDA approved for NOMID in the US.

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.

Anakinra (Kineret) was also approved in 2020 for use in DIRA.

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.

Anakinra Drug Information

Current FDA prescribing info: https://www.kineretrx.com/pdf/Full-Prescribing-Information-English.pdf