MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients
MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients
The purpose of this Cohort Treatment Plan is to allow access to crizanlizumab (SEG101) for
 eligible patients diagnosed with sickle cell disease (SCD) to prevent or reduce the frequency
 of vaso-occlusive crises (VOC). The patient's Treating Physician should follow the suggested
 treatment guidelines and comply with all local health authority regulations.
Inclusion criteria

 Written patient informed consent must be obtained prior to start of treatment.

 -  Male or female, 18 to 70 years of age (inclusive) on the day of informed consent
 signature.

 -  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).

 -  History of reoccurring VOC as assessed by the Treating Physician.

 -  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.

 -  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.

 -  Patient must meet the following laboratory values prior to treatment:

 -  Absolute Neutrophil Count ≥1.0 x 109/L

 -  Platelets ≥75 x 109/L

 -  Hemoglobin (Hgb) ≥4.0 g/dL

 -  Estimated glomerular filtration rate ≥ 45 mL/min

 -  Direct (conjugated) bilirubin ≤2.0 x ULN

 -  Alanine transaminase (ALT) ≤ 3.0 x ULN

 -  ECOG performance status ≤ 2

 Exclusion criteria

 Patients eligible for this Treatment Plan must not meet any of the following criteria:

 -  Is on a chronic transfusion program as defined by participating in a scheduled
 (pre-planned) series of transfusions (simple or exchange). Episodic transfusions are
 permitted.

 -  Contraindication or hypersensitivity to any drug or metabolites from similar class as
 crizanlizumab drug or to any excipients of the drug formulation.

 -  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.

 -  Use of therapeutic anticoagulation (prophylactic doses permitted) or antiplatelet
 therapy (other than aspirin or NSAIDs) within the 10 days prior to starting treatment

 -  Patient has no acute pathologic processes.

 -  Received a monoclonal antibody or immunomodulatory agent within 1 year of starting
 treatment, or has documented immunogenicity to a prior biologic.

 -  Pregnant or nursing women

 -  Patients with bleeding disorders

 -  Known history of testing positive for Human Immunodeficiency Virus (HIV) infection

 -  Patients with active Hepatitis B infections (HBsAg positive)

 o Note: Patients with antecedent but no active Hepatitis B (i.e. anti-HBc positive,
 HBsAg and HBV-DNA negative) are eligible

 -  Significant active infection or immune deficiency (including chronic use of
 immunosuppressive drugs)

 -  Has a serious mental or physical illness, which, in the opinion of the Treating
 Physician would compromise compliance to treatment.

 -  QTcF ≥470 msec prior to treatment

 Other protocol-defined inclusion/exclusion criteria may apply