FDA Grants Breakthrough Status to Novartis Investigational Drug

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Serelaxin, to treat acute heart failure, is the second Novartis drug to get new designation from FDA.

FDA has granted breakthrough therapy designation status to Novartis’ RLX030 (serelaxin), an investigational treatment for patients with acute heart failure (AHF). The drug is the second investigational candidate tby Novartis to gain the new designation by FDA intended to expedite drug review.

FDA concluded that RLX030 qualified for a breakthrough therapy designation after considering the available clinical evidence that supported a substantial improvement over currently available therapies for AHF, according to a Novartis statement.

RLX030 is being assessed by other health authorities, including the European Medicines Agency, for the treatment of AHF. RLX030 is a form of a naturally occurring hormone (human relaxin-2), present in both men and women. RLX030 is proposed for administration on admission to the emergency room to patients experiencing an AHF episode and is infused over a 48- hour period in addition to conventional therapies.


RLX030 is the second breakthrough therapy designation by FDA for a Novartis investigational treatment. In March 2013, Novartis received breakthrough therapy status for LDK378, a selective inhibitor of a cancer target called anaplastic lymphoma kinase. The drug is designed to treat patients with anaplastic lymphoma kinase positive (ALK+) metastatic nonsmall cell lung cancer, who had progressed during treatment with, or were intolerant to, crizotinib. Novartis has initiated two Phase II clinical trials to further evaluate LDK378 in this patient population with plans to initiate several Phase III clinical trials later this year. The first regulatory filing is anticipated by early 2014.

Breakthrough therapy designation is intended to expedite the development and review of drugs that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. The designation includes all of the fast-track program features as well as more intensive FDA guidance. The breakthrough therapy designation is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met. The Food and Drug Administration Safety and Innovation Act of 2012 included a provision that allows sponsors to request that their drug be designated as a breakthrough therapy, according to FDA information.