Lomitapide: a novel drug for homozygous familial hypercholesterolemia

Autor: Maurizio Averna, Maria D. Panno, Angelo B. Cefalù
Přispěvatelé: Panno, MD, Cefalu', AB, Averna, M
Rok vydání: 2014
Předmět:
Zdroj: Clinical Lipidology. 9:19-32
ISSN: 1758-4302
1758-4299
DOI: 10.2217/clp.13.74
Popis: 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.
Databáze: OpenAIRE
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