Novartis has nabbed its second breakthrough drug designation from the FDA, raising the prospect that it could shave some time off the regulatory process for the heart drug serelaxin (RLX-030).
But not by much. Investigators read out mixed pivotal results on the drug 7 months ago and Novartis ($NVS) is already well along the regulatory process. And serelaxin was tapped long ago as one of Novartis's top late-stage efforts.
Last fall, the pharma giant cited the program as Exhibit A in making the case to investors that it has the late-stage drugs that will be needed to arrest a slide in sales revenue. In a pivotal study investigators tracked a 37% reduction in the rate of death among patients with acute heart failure. The drug--a synthetic version of the hormone relaxin that aids pregnant women--helped improve shortness of breath among patients over 5 days. 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.
As a result, estimates of peak sales potential are all over the map, ranging from around $1.5 billion all the way up to a starry-eyed $6 billion. Even the low end, though, would be a win for Novartis. Faced by generic competition for big franchises like Gleevec and Diovan, Novartis badly needs to set up a new round of franchises like this one.
In recent months the FDA has handed out a lineup of these breakthrough drug designations, with all but one going to a Big Pharma program. The agency has repeatedly vowed that it will throw its doors open to drug development teams that qualify, helping to cut time off the R&D process by guiding adaptive trial designs and pushing the review process. But when regulators hand the designation to a late-stage program like this, the FDA raises questions about just how valuable this can be--aside from providing some new boasting rights to the company. Novartis was already on the breakthrough drug list with the cancer drug LDK378.
"RLX030 is representative of Novartis' strong commitment to develop innovative treatments for patients in areas of significant unmet need," said David Epstein, the division head of Novartis Pharmaceuticals. "Commonly used medicines for AHF only improve the immediate symptoms, so the additional effect on survival observed with RLX030 offers hope to patients and physicians."
- here's the press release