BOSTON ( TheStreet) -- Fans of FDA advisory panels have a riveting week of action ahead. Starting Tuesday morning, outside experts invited by FDA will weigh the risks and benefits of three different drugs on three consecutive days. The recommendations that emerge from these FDA advisory panels will factor into the agency's approval decisions. NPS Pharmaceuticals ( NPSP) brings Gattex, a treatment for short bowel syndrome, in front of the Gastrointestinal Drugs Advisory Committee on Tuesday Oct. 16. On Wednesday Oct. 17, the Endocrinologic and Metabolic Drugs Advisory Committee convenes for two days of hearings to review drugs aimed at helping patients with a rare genetic disorder that causes extremely high levels of cholesterol to accumulate in their blood. The panel will review Aegerion Pharmaceuticals' ( AEGR) lomitapide on Wednesday followed on Thursday by mipomersen, a drug developed by Isis Pharmaceuticals ( ISIS) and the Genzyme unit of Sanofi ( SNY). I will be live-blogging all three FDA advisory panels with play-by-play and analysis in real time from the opening gavels all the way through the final votes.
You can sign up for email reminders for each panel below. NPS Pharmaceuticals: Short bowel syndrome (SBS) is a chronic and disabling condition that strikes people, who for various reasons including Crohn's disease, have about 50% of their lower intestines surgically removed. A majority of patients can adapt to lead normal lives. A smaller group of SBS patients never recover full functionality of their lower intestines, and a result, must spend the rest of their lives attached to an intravenous parenteral nutrition bag that provides the liquid nutrients they need to survive. Gattex is an analogue of GLP-2, a protein involved with intestinal growth and function. Administered via a shot under the skin, Gattex is designed to boost the ability of the intestine to absorb nutrients. On Friday, NPS Pharma shares rose 19% to $10.86 due to a largely positive FDA review of Gattex released ahead of Tuesday's panel. The FDA appears to be concur with NPS Pharma that Gattex provides a clinically meaningful benefit for SBS patients. The agency also appears unconcerned about three cases of cancer reported in patients treated with Gattex.
Cancer risk possibly attributable to Gattex as well as the clinical meaningfulness of reductions in intravenous feeding will be the two major issues tackled by the experts on Tuesday's FDA advisory panel. Aegerion Pharmaceuticals: Homozygous familial hypercholesterolemia (HoFH) is a rare and life-threatening genetic condition in which a person inherits damaged genes from both parents, rendering the body unable to clear LDL cholesterol -- the "bad" form of cholesterol -- from their bloodstream. HoFH patients, an estimated 3,000 in the U.S., can have LDL cholesterol levels four to ten times higher than normal people and cannot be reduced with conventional lipid-lowering drugs. As a result, these patients have severely clogged arteries that lead to heart attacks in their 20s and death in their 30s from cardiovascular disease. Aegerion's lomitapide is a once-daily pill that works by blocking a protein responsible for the production of very-low-density lipoproteins (VLDL), which are the precursors for LDL cholesterol. By inhibiting this protein, lomitapide prevents the liver and kidneys from secreting lipids into the bloodstream. In a phase III study, 29 HoFH patients treated with lomitapide for 26 weeks saw their LDL cholesterol levels reduced by 40%. These patients were all on a background of statin therapy and some were also on apheresis, a dialysis-like process that filters cholesterol from blood. Twenty-three of 29 HoFH patients continued on lomitapide therapy during a 78-week safety extension phase of the phase III study. At 78 weeks, these patients had a 38% reduction in LDL cholesterol and some were able to reduce their background lipid-lowering medications. Lomitapide causes a buildup of fat in the liver, elevated liver enzymes and gastrointestinal problems including diarrhea, nausea and vomiting. The drug's liver-related toxicities are of particular concern but will be weighed against the life-threatening nature of HoFH. Isis Pharmaceuticals and Genzyme: Mipomersen is a once-weekly injection that inhibits apo-B, a protein that carries forms of cholesterol, including LDL-cholesterol, throughout the bloodstream. Isis and Genzyme, a subsidiary of Sanofi, are developing mipomersen for broader range of high cholesterol-related disease than just HoFH -- heterozygous hypercholesterolemia, severe hypercholesterolemia and patients with high cholesterol at risk for heart disease.
Thursday's panel will only review mipomersen as a treatment for HoFH. In a phase III study, 34 patients treated with mipomersen for 26 weeks saw 25% reduction in LDL cholesterol compared to a 3% reduction in patients treated with a placebo. Like with lomitapide, fatty liver and elevated liver enzyme levels were worrisome side effects recorded in the mipomersen phase III trial. Injection site reactions were also reported by 76% of mipomersen patients. -- Reported by Adam Feuerstein in Boston. Follow @AdamFeuerstein