Late Tuesday the FDA came through with an approval for mipomersen (Kynamro), an antisense drug advanced by Isis and partnered with Sanofi's Genzyme. It's the second new drug approved recently for homozygous familial hypercholesterolemia, a rare and dangerous form of high cholesterol.
So what goes into landing on $295,000 as the annual cost of a drug that maybe only 3,000 patients a year will use? That is the price tag that NPS Pharmaceuticals ($NPSP) has put on the recently approved Gattex, a treatment for short bowel syndrome.
Our readers who regularly check our website know that we kept up with the news online. But for everyone else, here's a roundup of the regulatory actions you may have missed.
The FDA has approved Aegerion's new drug for homozygous familial hypercholesterolemia, clearing a path to begin commercialization as the biotech comes to a major turning point.
The tepid 9-6 vote from an FDA advisory panel left the new drug application looking relatively weak compared with a 13-2 vote for rival treatment lomitapide from Aegerion Pharmaceuticals.
Genzyme and its partners at Isis Pharmaceuticals ($ISIS) won a key endorsement today for mipomersen, an experimental therapy for homozygous familial hypercholesterolemia. But the tepid 9-6 outcome left the new drug application looking relatively weak compared with the 13-2 vote that greeted the rival drug lomitapide from Aegerion Pharmaceuticals yesterday.
A group of FDA advisers took a hard look at Aegerion's ($AEGR) new drug for homozygous familial hypercholesterolemia and concluded that the clear need for a new therapy outshined any safety concerns they might have concerning liver disease. In a lopsided vote, 13 of the outside experts voted to recommend an approval of the drug, with only two voting against.
This morning the regulatory and research team at Aegerion Pharmaceuticals were hunkered down preparing for Wednesday's FDA panel review of their new drug for rare cases of homozygous familial hypercholesterolemia, encouraged by an FDA review of lomitapide that focused considerable attention on the right kind of risk evaluation and mitigation strategy that could keep the risk/benefit profile in proper proportion.
Investors were cheered somewhat by the FDA's review of Aegerion's lead rare disease drug lomitapide in the lead-up to this week's meeting of a panel of outside experts. The agency's internal critique has just been posted, supporting the clinical efficacy argument that the company presented from its small Phase III pivotal study of homozygous familial hypercholesterolemia (HoFH) but fretting over the drug's potential impact on the liver--a red flag in safety reviews.
Cambridge, MA-based Aegerion Pharmaceuticals has cleared an important milestone.