Resounding rejection for Novartis’ heart failure drug

An FDA advisory committee has voted unanimously against approving a new heart failure treatment from Novartis.

Serelaxin (RLX030) is a highly novel treatment, and was granted Breakthrough Therapy designation status by the FDA in June 2013 because of its potential to treat heart failure, an area of high unmet medical need.

However the FDA’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) took a very dim view of the drug’s pivotal trial, which it said failed to provide evidence of efficacy in treating dyspnoea (shortness of breath) and the rate of heart failure.

The FDA does not always follow the advice of its committees, but looks unlikely to differ in this instance, which will pour cold water on serelaxin’s status as a potential blockbuster.

Novartis says it will continue with its ongoing development programme includes RELAX-AHF-2, a global phase III outcomes study of more than 6,300 patients. Results are expected to be ready in 2016, which could yield more compelling data. However concerns expressed by the FDA committee about weaknesses in the trial design will be hard to address at this stage.

European rejection

The FDA committee’s judgement chimes with that from its European counterpart the CHMP.

The European Medicine Agency’s expert committee recommended rejection of the drug in January, noting that the study results did not demonstrate a benefit for short-term relief of dyspnoea over up to 24 hours, and though it acknowledged some benefit was seen over five days, it concluded that it was not clear this had clinical relevance.

In addition to this, the CHMP also raised concerns about the trial design, relating to how effectiveness was analysed and how differences in background treatment between the two study arms may have influenced the results.

Breakthrough Designation – no easy ride

The FDA committee’s outright rejection of the drug confirms that the special Breakthrough designation confers so special privileges on drugs when it comes to proving safety and efficacy. At the same time, it also raises questions about the benefits and impact of the special status, which in theory allows for far greater communication and collaboration between a company and the FDA in ensuring designated drugs have the best chance of being approved.


Hep C treatment given Breakthrough Therapy status


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