Lomitapide has been evaluated in fourteen Phase I and eight Phase II clinical trials, as well as a pivotal Phase III clinical trial in HoFH completed in 2011. An extension study to assess long-term safety is ongoing. Over 900 patients have been treated with lomitapide as part of these clinical trials.The most frequent adverse events in the Phase III clinical trial were gastrointestinal, and were generally mild to moderate. These events typically decreased after the patients were established on the maximally tolerated dose. Elevations in liver enzymes and hepatic fat were also observed in the Phase III trial. Four patients experienced elevations in liver enzymes of between five times to eleven times the upper limit of normal. Hepatic fat increased from a baseline of 1% to 8.3% at week 26, and then stabilized through week 78.
FDA Advisory Committee Recommends Approval Of Lomitapide For Treatment Of Homozygous Familial Hypercholesterolemia (HoFH)
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