June 27th, 2013
Novel Heart Failure Drug from Novartis Gains ‘Breakthrough Therapy’ Designation from FDA
Serelaxin, the novel therapy under development for the treatment of acute heart failure, has received a “breakthrough therapy” designation from the FDA, according to Novartis, the company developing the drug. The designation, the FDA explains, “is intended to expedite the development and review of drugs for serious or life-threatening conditions” and requires “preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.” In addition to getting a speedier review process, the sponsor of a drug with the designation receives “more intensive FDA guidance” on the development program.
Serelaxin, also known as RLX030, could become the first significant new treatment for acute decompensated heart failure patients in a generation. The FDA action was based on promising results from the RELAX-AHF trial, which was presented at the American Heart Association meeting last November and published simultaneously in the Lancet. Patients who received serelaxin in the trial had a significant reduction in relief from dyspnea, one of the primary endpoints of the trial. But what really attracted the interest of the experts was the surprising finding of a small but statistically significant difference in mortality at six months in favor of serelaxin. The trial investigators acknowledged that the findings of a six-month survival benefit “for a drug given for 48 h with a moderate number of death events (107 total) raises the question of whether this benefit is due to chance and whether another, confirmatory trial should be done.” At the time, heart failure expert Milton Packer said that “if the mortality effect is true then this trial changes the way we do things.” But, he emphasized, ”the real question is whether the mortality difference seen in this trial is true and replicable.”
Serelaxin is a recombinant form of the naturally occurring human hormone relaxin-2, which has been found to help women adjust to the cardiovascular changes that occur during pregnancy.