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A 56-week phase 3 data for orphan drug lomitapide are consistent with 26-week data, showing that the drug significantly reduced low density lipoprotein cholesterol levels in patients with homozygous familial hypercholesterolemia, according to the drug?s manufacturer Aegerion Pharmaceuticals.
A 56-week phase 3 data for orphan drug lomitapide are consistent with 26-week data, showing that the drug significantly reduced low density lipoprotein cholesterol levels in patients with homozygous familial hypercholesterolemia, according to the drug’s manufacturer Aegerion Pharmaceuticals.
The trial, which began in March 2010, is a single-arm, open-label study being conducted at 11 sites in 4 countries to evaluate the efficacy and long-term safety of lomitapide as an oral, once-a-day treatment for reducing LDL in patients with HoFH.
After a 6-week run-in period on current lipid-lowering therapy to determine baseline measurements, 29 adult male and female patients with a mean age of 31 years and amean LDL-C of 336 mg/dL (352 mg/dL for completers), received ascending doses of lomitapide titrated over the first 26 weeks of the trial to a maximum tolerated dose of 60 mg/day. To date, 3 patients withdrew due to gastrointestinal adverse events and 3 more withdrew consent to participate. Of the remaining 23 patients, 13 were able to have their background lipid-lowering therapy reduced between weeks 26 and 56 of the study. Overall, the lipid profile at week 56 is consistent with the data at week 26, with LDL-C and triglyceride levels dropping substantially and HDL-C levels returning to baseline levels.
"These phase 3 filing data will be at the core of our anticipated NDA and MAA submissions, and we are pleased that it is consistent with week 26 data,” said Marc Beer, CEO of Aegerion Pharmaceuticals in a company press statement. “We will now turn our attention to ensuring that these submissions are completed in a high-quality and timely fashion.”