A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators

Inclusion Criteria:

- Male or female patients greater than or equal to 2 years of age

- Documented congenital disorder of red blood cells (e.g., β-thalassemia major, sickle
cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions

- Cannot be adequately treated with a locally approved iron chelator due to one of the
following reasons:

- Documented non-compliance, defined as having taken less than 50% of the
prescribed chelation therapy doses in the 12 months prior to study entry

- Contraindications, unacceptable toxicities and/or documented poor response to
locally approved iron chelators despite proper compliance

- History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red
blood cells (PRBC])

- Serum ferritin value greater than or equal to 1000 µg/L

- Ability to comply with all study-related procedures, medications, and evaluations

Exclusion Criteria:

- Ongoing treatment with another iron chelator (Any other iron chelation therapy must
be discontinued at least 24 hours prior to study entry.)

- Patients who meet the eligibility criteria for any other ongoing Novartis sponsored
clinical study protocol with deferasirox and who have geographic access to these
sites

- Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment
with deferasirox

- Serum creatinine above the upper limit of normal at screening.

- Patients with ALT ≥ 500 U/L at screening.

- Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to
baseline

- Pregnancy (as indicated by serum β-HCG pregnancy test at screening for all female
patients with the potential to become pregnant) and patients who are breastfeeding

- Patients treated with systemic investigational drug within 4 weeks prior to or with
topical investigational drug within 7 days prior to the baseline visit

Other protocol-defined inclusion/exclusion criteria may apply.