Milton Friedman once said that the FDA was “obsessed with safety.” Some view that as a badge of honor, others not so much.
Two recent FDA advisory committee votes should please both groups.
An FDA panel recently voted 9-6 to recommend approval for Genzyme’s mipomersen for homozygous familial hypercholesterolemia. The positive vote came despite serious hepatic and other risks -- as long as every effort is made to prevent off-label use. A key mitigating factor was disease severity.
Benefit/Risk at its finest.
A second recent adcomm vote (13-2 in favor of approval for Aegerion’s lomitapide for homozygous familial hypercholesterolemia) also points to a more “needs-focused” philosophy and the committee also recommended a REMS targeted off-label use.
Safe Use is the new normal.