Defending Mipomersen
Isis, Genzyme: Mipomersen's LDL benefits outweigh adverse events
Investors and analysts quickly assumed the market for mipomersen from Isis Pharmaceuticals Inc. and Genzyme Corp. would be limited to only the most severe forms of uncontrolled hypercholesterolemia when adverse events were reported in the first two Phase III trials. Now, top-line data from the two final studies reported last week have raised additional concerns that use of the drug may be further limited because of high dropout rates.
Isis and Genzyme say Wall Street is missing the point, because the benefits of substantial reductions in LDL-C that mipomersen has demonstrated in the target indications of homozygous familial hypercholesterolemia (hoFH) and severe heterozygous FH (heFH) far outweigh the theoretical risks associated with the kinds of elevations in liver alanine transaminase (ALT) that have been reported...