NOMID Efficacy

KINERET efficacy in NOMID patients

In a long-term study, KINERET was shown to reduce NOMID symptoms and inflammation1

KINERET was studied in a prospective, long-term, open-label study of 43 patients with NOMID, 0.7 to 46 years of age, treated for up to 60 months.

Overall diary symptom sum score (DSSS) and each of its symptoms rapidly improved and was maintained over 60 months1

  • The figure shows mean values for observed cases at all given time points. ITT observed cases (N=29)
    • Results for this endpoint were consistent across all subgroups including age, gender, presence of NLRP3/CIAS1 mutation, and disease phenotype
  • Treatment with KINERET also appeared to be associated with improvement of, or stability in, other NOMID disease manifestations, up to 60 months
    • Central nervous system inflammation
    • Audiogram
    • Visual acuity
  • NOMID patients who were withdrawn from KINERET therapy responded again when treatment resumed
  • For the 11 patients who went through a withdrawal phase, disease symptoms and key biomarkers of inflammation worsened after withdrawal and promptly responded to reinstitution of KINERET therapy
Line graph of individual disease symptom scores comprising the overall diary symptom sum score by visit from baseline to 60 months

Study Design: Clinical efficacy and safety were determined by a prospective, long-term, open-label, uncontrolled study in 43 NOMID patients 0.7 to 46 years of age. Primary endpoints included change in disease-specific DSSS, including fever, rash, joint pain, vomiting, and headache, as well as change in the levels of inflammatory biomarkers SAA, high-sensitivity CRP (hsCRP), and ESR. Initial KINERET dose was 1 mg/kg to 2.4 mg/kg body weight, adjusted to 0.5 mg/kg to 1 mg/kg increments. The maximum dose actually studied was 7.6 mg/kg/day. The average maintenance dose was 3 mg to 4 mg/kg daily. The duration of treatment with KINERET was up to 60 months.

Systemic inflammatory markers were significantly reduced in all patients treated with KINERET2

  • Systemic inflammatory remission was achieved in all patients; however, remission and relapse continued to occur in patients with infections or stress3
    • Inflammatory remission was defined as CRP level ≤0.5 mg/dL, ESR ≤25 mm/hour, and SAA ≤10 mg/liter3
  • Patients had significant reductions in inflammatory markers from baseline at 6, 12, and 36 months and results were stable from 36 to 602,3
Bar graph of inflammatory markers from baseline to 6 months. The bars are grouped by measurements at baseline, 1 month, 3 months, and 6 months

Study design: Clinical efficacy and safety were determined by a prospective, long-term, open-label, uncontrolled study in 43 NOMID patients 0.7 to 46 years of age. Primary endpoints included change in disease-specific Diary Symptom Sum Scores (DSSS), including fever, rash, joint pain, vomiting, and headache, as well as change in the levels of inflammatory biomarkers SAA, hsCRP, and ESR. Initial KINERET dose was 1 mg/kg to 2.4 mg/kg body weight, adjusted to 0.5 mg/kg to 1 mg/kg increments. The maximum dose actually studied was 7.6 mg/kg/day. The average maintenance dose was 3 to 4 mg/kg daily. The duration of treatment with KINERET was up to 60 months.3

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Indications and Important Safety Information
INDICATION

KINERET® (anakinra) is an interleukin-1 receptor antagonist indicated for:

Rheumatoid Arthritis (RA). Reduction in signs and symptoms and slowing the progression of structural damage in moderately to severely active rheumatoid arthritis, in patients 18 years of age or older who have failed 1 or more disease-modifying antirheumatic drugs (DMARDs)

Cryopyrin-Associated Periodic Syndromes (CAPS). Treatment of Neonatal-Onset Multisystem Inflammatory Disease (NOMID)

Deficiency of Interleukin-1 Receptor Antagonist (DIRA). Treatment of Deficiency of Interleukin-1 Receptor Antagonist (DIRA)

CONTRAINDICATION

KINERET is contraindicated in patients with known hypersensitivity to E. coli–derived proteins, KINERET, or to any components of the product

IMPORTANT SAFETY INFORMATION

Serious Infections. In RA, discontinue use if serious infection develops. In KINERET-treated NOMID or DIRA patients, the risk of a disease flare when discontinuing KINERET treatment should be weighed against the potential risk of continued treatment. Do not initiate KINERET in patients with active infections

Use in combination with Tumor Necrosis Factor (TNF)-blocking agents is not recommended

Hypersensitivity reactions, including anaphylactic reactions and angioedema, have been reported. Patients with DIRA may have an increased risk of allergic reactions, particularly in the first several weeks after starting KINERET treatment

Immunosuppression. The impact of treatment with KINERET on active and/or chronic infections and the development of malignancies is not known

Immunizations. Live vaccines should not be given concurrently with KINERET

Neutrophil counts should be assessed prior to initiating KINERET treatment, and while receiving KINERET, monthly for 3 months, and thereafter quarterly for a period up to 1 year

Serious Adverse Reactions

RA: The most serious adverse reactions were: Serious Infections and Neutropenia, particularly when used in combination with TNF blocking agents.

NOMID and DIRA: The most serious adverse events were infections.

Most Common Adverse Reactions

RA: The most common adverse reactions (incidence ≥ 5%) are injection site reaction, worsening of rheumatoid arthritis, upper respiratory tract infection, headache, nausea, diarrhea, sinusitis, arthralgia, flu-like symptoms, and abdominal pain

NOMID: The most common AEs during the first 6 months of treatment (incidence > 10%) are injection site reaction, headache, vomiting, arthralgia, pyrexia, and nasopharyngitis

DIRA: The most common AEs are upper respiratory tract infections, rash, pyrexia, influenza-like illness, and gastroenteritis

Post-marketing Experience

Hepato-biliary disorders (elevations of transaminases; non-infectious hepatitis) and thrombocytopenia, including severe thrombocytopenia have been identified during postapproval use of KINERET. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.

These are not all the possible risks associated with KINERET. Please see Full Prescribing Information for KINERET at https://www.kineretrx.com/hcp/
To report suspected adverse reactions, contact Sobi North America at 1-866-773-5274 or FDA at 1-800-FDA-1088
Click here for full Prescribing Information for KINERET.

References: 1. KINERET [Prescribing Information]. Stockholm, Sweden: Swedish Orphan Biovitrum AB (publ). 2. Goldbach-Mansky R. Current status of understanding the pathogenesis and management of patients with NOMID/CINCA. Curr Rheumatol Rep. 2011;13(2):123-131. 3. Sibley CH, Plass N, Snow J, Wiggs EA, et al. Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: a cohort study to determine three- and five-year outcomes. Arthritis Rheum. 2012;64(7):2375-2386.