AstraZeneca heart drug denied US approval

AstraZeneca (AZ) has received a complete response letter from the US Food and Drug Administration (FDA) stating that the new drug application for Brilinta (ticagrelor), a potential new drug for acute coronary syndrome, cannot be approved in its current form. 

The FDA did not request additional studies, but did ask for additional analyses of the data from the company's PLATO trial, which enrolled 18,624 patients in 43 countries to test whether ticagrelor could achieve clinically meaningful cardiovascular and safety endpoints in ACS patients above and beyond those afforded by Bristol-Myers Squibb/sanofi-aventis's Plavix (clopidogrel).

The study demonstrated that Brilinta was generally more effective in the prevention of cardiovascular events in patients with ACS than Plavix, the world's second best-selling drug. However, US patients were more likely to experience heart problems with Brilinta.

"AstraZeneca is evaluating the contents of the CRL and will respond to the agency's request for additional analyses of the PLATO data as soon as possible," the company said in a statement. "The company remains confident in the NDA submission for ticagrelor and in its ability to respond to the agency's questions."

AZ submitted the NDA in November 2009. The original The Prescription Drug User Fee Act (PDUFA) date for FDA action on the application was September 16, but the agency pushed it back by three months, fueling speculation that trouble might be ahead for the drug.

Brilinta, an oral tablet, was granted marketing authorisation under the tradename Brilique by the European Commission earlier this month. In August, an advisory committee to the FDA voted 7-1 in favour of recommending that the agency approve the drug.

According to analysts' opinions collated by Thomson Reuters, Brilinta could achieve sales of $1.2bn by 2014 and grow from there.