Novartis AG’s experimental heart failure medication failed to win the backing of U.S regulatory advisers, creating a potential hurdle to the company’s plan to build a portfolio of cardiac therapies around the drug.
An 11-member advisory panel to the Food and Drug Administration voted unanimously today that the product, serelaxin, shouldn’t be cleared for sale. The FDA doesn’t have to follow the advice of its advisers and is scheduled to rule on the therapy’s approval by May 17.
FDA staff determined there is insufficient evidence that serelaxin will benefit patients, in a report posted March 25. The drug, similar to the hormone relaxin that protects the hearts of pregnant women, failed to win backing from European Union regulators in January who questioned the drug’s efficacy. Serelaxin could generate $523 million in sales in 2018, based on the average of eight analysts’ estimates compiled by Bloomberg.
“I think this was more a failure of trial design than it was of the drug itself,” said Stuart Rich, a panel member and professor at the University of Chicago Pritzker School of Medicine. “A drug that will reduce morbidity, mortality, length of stay is really needed and hopefully these hypotheses in this trial will be proven out”
The panel, like the FDA staff, was concerned that Novartis based their application on a single study that sought to primarily measure the drug’s effect on labored breathing. The data is inadequate to determine whether serelaxin prevents the disease from getting worse.
The Basel, Switzerland-based drugmaker needs to make up for anticipated revenue losses as the heart medicine Diovan and cancer treatment Gleevec, its biggest sellers, start to face generic competition. The two made up about $8.2 billion of Novartis’s $58 billion in 2013 revenue.
“What they did observe was an effect on worsening heart failure, probably real, but hard to define because it was defined so many different ways and there are so many arbitrary parts,” Rich said. “If they can, in a subsequent trial, better design it so we know the magnitude of that effect that would be ideal.”
Novartis eventually wants to gain FDA approval of serelaxin to reduce death from heart failure, said Rob Kowalski, global head of drug regulatory affairs at Novartis. The FDA granted serelaxin breakthrough status for the use, meaning the agency considers it a likely substantial improvement over current therapies and will expedite review of the drug.
Novartis expects to complete a study on mortality by the end of 2016, Kowalski said.
“The discussion provided important information that we will address with the FDA as it completes its review,” said Tim Wright, global head of development at Novartis. “In the meantime we’ll continue to drive our robust clinical trial program and build upon the already established body of evidence.”
About 5 million people in the U.S. suffer from heart failure and about 1 million of those are hospitalized each year, Kowalski said. With serelaxin, Novartis plans to target those who end up in the hospital.
Heart failure occurs when the heart can’t pump enough blood to the body. Doctors have relied on nitrates and diuretics to relax the heart’s blood vessels and reduce fluid build-up. Serelaxin was tested in addition to the standard of care.
Novartis is re-applying for conditional approval from the European Union that will require another review once the trial measuring whether serelaxin reduces deaths is complete.