Isis earns $25 million milestone from Genzyme as US FDA accepts Kynamro NDA

The US Food and Drug Administration has accepted for filing the New Drug Application for Kynamro (mipomersen sodium), under development by French drug major Sanofi’s (Euronext: SAN) Genzyme subsidiary and Isis Pharmaceuticals (Nasdaq: ISIS) for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). The NDA filing with the FDA triggers a $25 million milestone payment to Isis from Genzyme.

The NDA filing with the FDA represents a significant achievement in the development of Kynamro and our efforts to get this important new drug to the market for patients who are at high-risk of a cardiovascular event,” said David Meeker, president and chief executive of Genzyme.

Genzyme is preparing for the European Union and US launch of Kynamro in 2012 and is also looking to commercialize Kynamro in other major markets. Kynamro enjoys orphan drug status in the US for the HoFH indication. According to Genzyme, about 40,000 patients in the US and Europe suffer from HoFH and severe heFH. If Kynamro proves successful, it would give Isis and Genzyme a potentially large share of the $35 billion annual global market for cholesterol-lowering drugs. However, how big a chunk depends on the extent of uses for which Kynamro is ultimately approved.