Promising Heart Failure Drug Receives ‘Breakthrough' FDA Designation

The US Food and Drug Administration has awarded “breakthrough therapy” status to a synthetic copy of a naturally occurring human hormone for the treatment of acute heart failure (AHF), drug developer Novartis announced today.

The Switzerland-based pharmaceutical company said the FDA’s recognition is backed by phase 3 clinical trial data presented at the American Heart Association’s 2012 Scientific Sessions that showed its investigational serelaxin therapy, also known as RLX030, reduced cardiovascular death and all-cause mortality by 37 percent at six months in a study group of 1,161 acute heart failure patients. Taking the recent “breakthrough” status of Novartis’s experimental lung cancer treatment compound into account, serelaxin is the company’s second promising treatment to receive the designation since July 2012, when the FDA formally introduced the program as a more rapid development and approval path for drugs that demonstrate “substantial improvement on existing therapies for clinically significant endpoints.”

Novartis’s man-made version of human relaxin-2 hormone, whose levels increase in pregnancy to manage additional cardiovascular strains, has been proposed for administration in patients experiencing an AHF episode on admission to an emergency room and infusion with conventional therapies over a two-day period, the company said. In a statement, Novartis Pharmaceuticals division head David Epstein noted that “commonly used medicines for AHF only improve the immediate symptoms, so the additional effect on survival observed with RLX030 offers hope to patients and physicians.”

According to 2012 guidelines from the European Society of Cardiology, more than 15 million patients experience heart failure globally, and since AHF episodes require urgent treatment, the condition is currently the most common cause of hospitalization in patients over the age of 65. If approved by the FDA, serelaxin may constitute the first pharmaceutical breakthrough for AHF patients in two decades, Novartis said.