Genzyme Corp. and its partner Isis Pharmaceuticals Inc. (NASDAQ: ISIS), said the U.S. Food and Drug Administration has accepted their potential treatment for a rare, genetic form of extremely high cholesterol for review, after it was initially sent to the agency in March. The acceptance by the FDA triggers a $25 million milestone payment to Isis from Cambridge, Mass.-based Genzyme, which is owned by French drug maker Sanofi (NYSE: SNY).
Although the potential drug, which would be marketed as Kynamro if approved, serves a rare-disease population, the FDA informed the companies that it will not receive an accelerated, six-month review. A decision date has been set for Jan. 29, 2013. The companies are also awaiting a decision from EU regulators, who have been reviewing the drug candidate since a new drug application (NDA) was submitted in July 2011.
The disease, a form of hypercholesterolemia, is life-threatening for patients, as it causes heart attacks and strokes.
“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,” Genzyme CEO Dr. David Meeker, said in a statement. “We look forward to continuing the review process with the U.S. and EU regulatory authorities to bring Kynamro to patients in need.”
Another Cambridge, Mass.-based biotech, Aegerion Pharmaceuticals (Nasdaq: AEGR), sent a rival potential therapy to the FDA, also in March. Aegerion also received a standard, 10-month review for its drug candidate, lomitapide.
