Prospects for Novartis’ acute heart failure (AHF) drug serelaxin look increasingly dim after FDA reviewers recommended against approval ahead of an advisory committee meeting tomorrow.
The reviewers recommend (PDF) that the drug “not be approved at this time because there is insufficient evidence to support the proposed indication”, which is to improve symptoms and reduce the progression of AHF.
The negative verdict by the FDA staffer comes after the European Medicines Agency’s main advisory panel – the Committee for Medicinal Products for Human Use (CHMP) – also said it was unable to back the drug as a treatment for AHF because of a lack of clinical evidence.
Like the CHMP, FDA reviewers Melanie Blank and Tzu-Yun McDowell are concerned there is only one trial supporting serelaxin’s clinical benefits in AHF, namely the RELAX-AHF study which found that the drug was able to improve shortness of breath (dyspnoea), and also points out that the validity of the finding is open to debate from a statistical viewpoint.
Novartis has always maintained that its decision to press on with filing despite having only one study was predicated in the fact that there has been no treatment advance in AHF for more than two decades. For that reason, the FDA awarded serelaxin breakthrough status last year.
The reviewers are also unhappy that Novartis is pursuing a broader claim than just improving dyspnoea, arguing there is no evidence that serelaxin can reduce the rate of worsening AHF. On the plus side for the sponsor, they do suggest that the safety profile of serelaxin is acceptable.
It is possible that the FDA’s advisory committee may take a different stance on the drug, but the prevailing regulatory sentiment seems to be that Novartis will have to carry out another trial of serelaxin before it can mount a convincing case for approval.
Prior to the CHMP’s decision last year analysts had been predicting peak sales potential for serelaxin of between $500m and $1.5bn. Novartis has remained bullish on the drug’s prospects, and said earlier this year it will seek conditional approval in the EU while an ongoing analysis of mortality data is completed.