Serelaxin for AHF Gets 'Breakthrough-Therapy' Path Through FDA

Shelley Wood

July 01, 2013

BASEL, Switzerland — The FDA has granted "breakthrough therapy" designation to serelaxin (Novartis Pharmaceuticals), the novel recombinant form of human relaxin 2 that has demonstrated promising clinical findings in acute heart failure (AHF) [1].

The designation, announced by the drug maker early this morning, means the experimental drug, also known as RLX030, likely faces a speedier regulatory path through the US approval process.

As previously reported by heartwire , serelaxin reduced shortness of breath as assessed using one of two dyspnea end points and appeared to reduce all-cause and cardiovascular mortality, as well as signs and symptoms of congestion and worsening heart failure in the Relaxin for the Treatment of AHF (RELAX-AHF) trial. Relaxin is the hormone that modulates the cardiovascular responses during pregnancy by increasing vasodilation and renal function.

The human-made form, serelaxin, was one of the major talking points at last year's American Heart Association meeting, where heart-failure experts expressed excitement at the prospect of a new drug in a field that has not seen new therapies in over 20 years. Some, however, expressed concerns that RELAX-AHF only met one of its two co–primary dyspnea end points and did not meet statistical significance on its two secondary end points: days alive at day 60 and a composite of cardiovascular death and heart-/renal-failure hospitalizations up to day 60.

Overall, there was a significant 37% reduction in risk of all-cause mortality at six months, as well as a significant 37% reduction in risk of cardiovascular mortality with serelaxin; both were prespecified end points. Observers at the AHA meeting expressed surprised that a drug could reduce deaths without cutting down on the number of hospitalizations.

The FDA clearly believes the drug holds the potential to help a difficult-to-treat patient group. Breakthrough designation therapy is reserved for the development or review of drugs seen as potential game changers for serious and life-threatening conditions that have preliminary evidence indicating improvement in at least one clinically significant end point over other available therapies.

The fast tracking that accompanies this designation includes regular meetings, "timely" advice, and interactive communication between the FDA and the sponsor, help with clinical-trial design, a stepped-up review process, and the involvement of senior managers and experienced reviewers at the FDA.

An earlier version of this story incorrectly stated that all-cause mortality and cardiovascular mortality were not prespecified end points.

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