Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

This was a phase III open label clinical trial to evaluate the long-term efficacy and safety
of lomitapide at the maximum tolerated dose (for each patient) established during the
clinical trial 733-005/UP1002. Subjects completing the 78-week treatment period in study
733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate
in 733-012. The treatment period continued until a decision was made by the local competent
authority regarding marketing authorization. Lomitapide will be given orally once daily.
Patient specific doses will be carried forward from 733-005/UP1002, but will not exceed the
maximum tolerated dose the patient received during 733-005 /UP1002. The maximum dose for any
patient was 80 mg/day. There was no reference therapy in this trial. The effects of the study
drug were compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy
including plasmapheresis or LDL apheresis is permitted.

Note that hereafter, Week 0 in Study 733-005/UP1002 will be referred to as Baseline and Week
48 in Study AEGR-733-012 will be referred to as Week 126 (of overall treatment).

Inclusion Criteria:

1. Completed UP1002 or 733-005.

2. Willing and able to provide consent and comply with the requirements of the study
protocol.

Exclusion Criteria:

1. Met any of the stopping rules for study discontinuation at the final visit of study
UP1002 or 733-005.