Exceptional Access Program (EAP)

Canakinumab

Brand(s)

• Ilaris

Dosage Form/Strength

• 150 mg/vial injection

Indications

Muckle-Wells Syndrome (MWS)

The patient has a diagnosis of Muckle-Wells Syndrome (MWS) based on meeting each of the following confirmatory results:

1. Clinical diagnosis of MWS;
2. NLRP3 mutation (mutational analysis required);
3. SAA levels ≥ 10 mg/L; and
4. Assessment score of patient’s disease activity determined by the following 4 parameters:
   • Skin disease
   • Arthralgia
   • Conjunctivitis
   • Fatigue/malaise ≥ mild, moderate, or severe.

If a patient is without NLRP3 mutation, a request will be considered on a case-by-case basis if the other confirmatory criteria are met and if the exclusion criteria are not met. However, the initial approval would be for 6 months; if the patient demonstrated a sustained SAA level < 10 mg/L, a renewal of funding for a 1-year approval period would be considered.

• Initial Approval Period: 1 year

NOMID Syndrome

The patient has a diagnosis of Neonatal-Onset Multisystem Inflammatory Disease (NOMID) based on meeting each of the following confirmatory results:

1. Clinical diagnosis of NOMID.
2. Presentation of symptoms of NOMID in a patient < 6 months of age.
3. NLRP3 mutation (mutational analysis required).
4. A score of patient’s disease activity determined by the following 4 parameters:
   • Skin disease
   • Arthralgia
   • Conjunctivitis
   • Fatigue/malaise ≥ mild, moderate, or severe.

If a patient is without NLRP3 mutation, a request will be considered on a case-by-case basis if the other confirmatory criteria are met and if the exclusion criteria are not met. However, the initial approval would be for 6 months; if the patient demonstrated a sustained SAA level < 10 mg/L, a renewal of funding for a 1-year approval period would be considered.

• Initial Approval Period: 6 months

For Both Indications

• Supervision: For all patients, the treatment is supervised by medical specialists with knowledge in the management of CAPS (Cryopyrin-Associated Periodic Syndromes).

Approved Dosage

• Bodyweight > 40 kg:
  • 150 mg subcutaneously every 8 weeks
• Bodyweight = 15 kg – 40 kg:
  • 2 mg/kg subcutaneously every 8 weeks
  • (For children 15 kg – 40 kg with an inadequate response, the dose can be increased to 3 mg/kg.)

Exclusion Criteria

Funding requests will be denied if any of the following exclusion criteria are met:

i) Bedridden State:

• The patient is bedridden where any physical activity brings on discomfort and symptoms which occur at rest AND not amenable to surgical/medical intervention;

ii) Other Life-Threatening Diseases:

• The patient has another life-threatening disease where prognosis is unlikely to be influenced by Ilaris® (canakinumab) therapy (e.g., neuroblastoma, leukemia, etc.);

iii) Limited Life Expectancy:

• The patient has a life-expectancy of six months or less, regardless of the cause; or

iv) Familial Cold Auto-Inflammatory Syndrome (FCAS).

Renewal Criteria

Confirmation from the Patient’s Physician that:

1. Benefit from Therapy:
   • The patient has benefited or continues to benefit from therapy with Ilaris, is expected to continue to do so, and each of the following is met:
2. SAA Levels:
   • SAA levels < 10 mg/L.
   • If SAA levels are 10 mg/L or higher over a 6-month interval and sustained over two consecutive 6-month intervals, approval for funding may be withdrawn.
3. Assessment Score:
   • An assessment score of patient’s skin disease, arthralgia, conjunctivitis, and fatigue/malaise shows no or minimal disease activity.
4. Additional Patient Conditions:
   • a) The patient is not bedridden where any physical activity brings on discomfort and symptoms which occur at rest AND not amenable to surgical/medical intervention;
   • b) The patient has no other life-threatening disease where prognosis is unlikely to be influenced by Ilaris® (canakinumab) therapy (e.g., neuroblastoma, leukemia, etc.);
   • c) The patient has not developed a life-threatening complication or a severe injection reaction to Ilaris® (canakinumab) not treatable by other therapeutic measures;
   • d) The patient has adhered with prescribed injection schedule for optimal management of the disease;
   • e) The patient has adhered to all safety and effectiveness monitoring of the treatment;
   • f) Treatment will be supervised by medical specialists with knowledge in the management of CAPS.

• Approved Dosage for Subsequent Approvals: Same as Initial Approvals.

Duration of Approval

• Initial Approval Period:
  • Muckle-Wells Syndrome (MWS): 1 year
  • NOMID Syndrome: 6 months
• Renewals:
  • For MWS:
    • Renewal Period: 1 year (after initial 1-year approval)
  • For NOMID:
    • Renewal Period: 1 year (after initial 6-month approval)
• For Subsequent Renewals:
  • Same as Initial Approvals