Another hurdle for Serelaxin 

The Pumping Marvellous Foundation
The Pumping Marvellous Foundation – the UK’s heart failure charity

In advance of an upcoming advisory committee meeting, the FDA in-house review says point blank that the application for the heart drug serelaxin should be rejected as Novartis has yet to provide the decisive data on efficacy needed to green light the therapy.

The review states that Novartis didn’t back up the proposed indication on acute heart failure, didn’t conduct a second trial, or design the single study to provide the equivalent data load of two studies to confirm the drug’s effect on dyspnea (breathlessness), and didn’t actually engineer the pivotal trial to back up its claims on easing symptoms and changing the rate of heart failure in any case.

“We recommend that serelaxin not be approved at this time because there is insufficient evidence to support the proposed indication: to “improve the symptoms of acute heart failure through reduction of the rate of worsening of heart failure,”‘ and noted the review by Melanie Blank and Tzu-Yun McDowell. “We did not identify any significant safety concerns precluding approval. In particular, the favorable 180-day mortality data for serelaxin is reassuring.”

Serelaxin is a synthetic version of the hormone relaxin that aids pregnant women–helped improve shortness of breath among patients over 5 days, the company asserted. But the drug missed other endpoints, raising fears among analysts that an approval this year could simply set up a more critical review process among payers.

Back in January the European Medicines Agency‘s Committee for Medicinal Products for Human Use rejected Novartis’ application for serelaxin, citing the development team for failing to demonstrate quick relief for heart disease in the first 24 hours, failing to demonstrate the significance of the benefit over 5 days and calling into question the data that was presented. Novartis immediately shifted tactics, though, saying it would shoot for a conditional approval.

The FDA has however blessed serelaxin with breakthrough drug designation status last summer. The BTD title was bestowed during the first big wave in the months after the agency launched the effort to speed up the development of the industry’s most promising therapies. Novartis is now gathering mortality data on the drug, but a spokesperson told Bloomberg that the data wouldn’t be available until 2016.

The FDA wrote: “The results of RELAX-AHF were driven by worsening heart failure that could be easily medically managed with small increases in doses of IV diuretics. In fact, the mean difference in IV furosemide doses between treatment groups over the 5-day assessment period was 60 mg and the median difference was 20 mg (higher in the placebo group). The small difference in diuretic use provides some evidence of drug activity but it is a small treatment effect seen in one trial.”

The FDA advisory committee meets on Thursday.

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