Lomitapide (Juxtapid® and Lojuxta®; Aegerion Pharmaceuticals, Inc., MA, USA), an orally administered inhibitor of the microsomal triglyceride transfer protein, inhibits the synthesis and secretion of ApoB-containing lipoproteins and, thus, reduces plasma levels of LDL cholesterol (LDL-C). Lomitapide has been approved for the therapy of homozygous familial hypercholesterolemia patients. After a proof-of-concept Phase II trial, lomitapide has been tested in a multinational single-arm, open-label, 78-week, Phase III trial. Lomitapide effectively reduced mean plasma LDL-C levels by 50% from baseline in 23 adults with homozygous familial hypercholesterolemia over a 26-week treatment period and this reduction was sustained for an additional 52 weeks of lomitapide treatment. The Phase III trial also demonstrated that 46% of patients (six out of 13) interrupted or reduced the frequency of apheresis treatments because of an important and stable reduction of LDL-C. Lomitapide was generally well tolerated and the most common adverse events in the Phase III trial were gastrointestinal and hepatic events.

Panno, M.D., Cefalu', A.B., & Averna, M. (2014). Lomitapide: a novel drug for homozygous familial hypercholesterolemia. CLINICAL LIPIDOLOGY, 9(1), 19-32 [10.2217/clp.13.74].

Lomitapide: a novel drug for homozygous familial hypercholesterolemia

CEFALU', Angelo Baldassare;AVERNA, Maurizio
2014

Abstract

Lomitapide (Juxtapid® and Lojuxta®; Aegerion Pharmaceuticals, Inc., MA, USA), an orally administered inhibitor of the microsomal triglyceride transfer protein, inhibits the synthesis and secretion of ApoB-containing lipoproteins and, thus, reduces plasma levels of LDL cholesterol (LDL-C). Lomitapide has been approved for the therapy of homozygous familial hypercholesterolemia patients. After a proof-of-concept Phase II trial, lomitapide has been tested in a multinational single-arm, open-label, 78-week, Phase III trial. Lomitapide effectively reduced mean plasma LDL-C levels by 50% from baseline in 23 adults with homozygous familial hypercholesterolemia over a 26-week treatment period and this reduction was sustained for an additional 52 weeks of lomitapide treatment. The Phase III trial also demonstrated that 46% of patients (six out of 13) interrupted or reduced the frequency of apheresis treatments because of an important and stable reduction of LDL-C. Lomitapide was generally well tolerated and the most common adverse events in the Phase III trial were gastrointestinal and hepatic events.
Settore MED/09 - Medicina Interna
Panno, M.D., Cefalu', A.B., & Averna, M. (2014). Lomitapide: a novel drug for homozygous familial hypercholesterolemia. CLINICAL LIPIDOLOGY, 9(1), 19-32 [10.2217/clp.13.74].
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/10447/98662
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