MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients

Inclusion criteria

Written patient informed consent must be obtained prior to start of treatment, including
all necessary consents (or their legal representatives, where applicable). 1. Male or female, 16 years of age (inclusive) or older on the day of informed consent signature. 2. Confirmed diagnosis of sickle cell disease by hemoglobin electrophoresis or high performance liquid chromatography (HPLC) [performed locally]. All sickle cell disease genotypes are eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others). 3. History of recurrent VOC as assessed by the Treating Physician. 4. Patients receiving HU/HC, L-glutamine (Endari), erythropoietin stimulating agents or other therapies as prevention therapy and continue to experience VOC while on any of these treatments. • Patients can continue taking the preventive therapy. 5. Patient is not a candidate to be treated with alternative treatment options or has discontinued alternative treatments due to unacceptable benefit risk as documented by the Treating Physician. 6. Patient must meet the following laboratory values prior to treatment: - Absolute Neutrophil Count ≥1.0 x 10^9/L - Platelets ≥ 75 x 10^9/L - Hemoglobin (Hgb) ≥ 4.0 g/dL - Estimated glomerular filtration rate ≥ 45 mL/min/1.73 m2 using CKD-EPI formula - Direct (conjugated) bilirubin ≤ 2.0 x ULN - Alanine transaminase (ALT) ≤ 3.0 x ULN 7. ECOG performance status ≤ 2.0 8. Received standard age-appropriate care for SCD, including an up-to-date record of immunizations, as per local requirements 9. Patients who are clinically stable and are in a non-crisis state at the time of treatment start

Exclusion criteria

Patients eligible for this Treatment Plan must not meet any of the following criteria: 1. Contraindication or hypersensitivity to any drug or metabolites from similar class as crizanlizumab drug or to any excipients of the drug formulation. 2. History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the Treating Physician may pose an increased risk of serious infusion reaction. 3. Received a monoclonal antibody or immunomodulatory agent within 1 year of starting treatment, or has documented immunogenicity to a prior biologic. 4. Pregnant or nursing women 5. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 15 weeks after stopping treatment. Highly effective contraception methods include: - Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception - Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking crizanlizumab. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment - Male sterilization (at least 6 months prior to treatment). The vasectomized male partner should be the sole partner for that patient - Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate