Isis Antisense Drug Wins FDA Approval

The Food & Drug Administration has granted Isis Pharmaceuticals and its partner Genzyme approval to market Kynamro, a once-weekly subcutaneous injection to lower lipid levels in people with a rare condition called homozygous familial hypercholesterolemia (HoFH). Despite the drug’s tiny market—HoFH strikes just one in a million people—its approval provides significant validation of Isis’ antisense technology platform. Kynamro is the first systemically administered antisense treatment to reach the market, an achievement 22 years in the making for Isis. With the approval, Isis scores a $25 million milestone payment from Genzyme.