Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload

Inclusion Criteria:

- Written informed consent/assent before any study-specific procedures. Consent will be
obtained from parent(s) or legal guardians. Investigators will also obtain assent of
patients according to local guidelines.

- Male and female children and adolescents aged ≥ 2 and < 18 years. [France: Male and
female children and adolescent aged ≥ 2 and < 18 years old, however children aged ≥ 2
and ≤ 6years can be enrolled only when deferoxamine treatment is contraindicated or
inadequate in these patients as per investigator decision. Applicable to core phase
only. Once in the core phase patients can turn 18 years and still be considered
eligible, also for participation in the optional extension phase.

- Any transfusion-dependent anemia associated with iron overload requiring iron
chelation therapy and with a history of transfusion of approximately 20 PRBC units and
a treatment goal to reduce iron burden (300mL PRBC = 1 unit in adults whereas 4 ml/kg
PRBC is considered 1 unit for children).

- Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the
mean value will be used for eligibility criteria).

- Patient has to have participated and completed the 48 weeks core phase treatment as
per protocol (For optional extension phase eligibility only).

Exclusion Criteria:

- Creatinine clearance below the contraindication limit in the locally approved
prescribing information (using Schwartz formula) at screening visit 1 or screening
visit 2.

- Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and or
screening Visit 2

- ALT and/or AST > 3.0 x ULN at screening visit 1 or screening visit 2.

- (Criterion no longer applicable, removed as part of Amendment 1): Prior iron chelation
therapy.

- Liver disease with severity of Child-Pugh class B or C.

- Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg
in a second morning urine sample at screening Visit 1 or screening Visit 2.

- Patients with significant impaired gastrointestinal (GI) function or GI disease that
may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases,
uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel
resection).

Other protocol-defined Inclusion/Exclusion may apply.