FDA formally rejects Aveo Pharmaceuticals application for approval of kidney cancer drug

Federal regulators have formally rejected Aveo Pharmaceuticals Inc.’s application to sell its kidney cancer drug in the US, the Cambridge company said Monday.

Aveo acknowledged it received a letter from the Food and Drug Administration alerting the company that the agency will not approve the new drug application for the experimental drug, called tivozanib, in its present form. The drug treats advanced renal cell carcinoma, a form of kidney cancer that kills tens of thousands of people around the world each year.

The rejection has been expected since an FDA advisory committee voted 13 to 1 on May 1 to recommended against the application, suggesting it was not clearly superior to existing renal cell carcinoma treatments. The agency had promised a decision by July 28.

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In their response letter, FDA officials shed some light on the reasoning of regulators, criticizing the way Aveo presented results from a late-stage clinical trial.

“Inconsistent progression-free survival results and imbalance in post-study treatments made the (tivozanib) results uninterpretable and inconclusive when making a risk-benefit assessment,” according to the company’s summary of the FDA letter. Aveo said the agency’s staff recommended, for the second time, that the company conduct a new clinical study.

But Aveo’s Japanese partner, Astellas Pharma Inc., already has said it will not finance a new trial of tivozanib to treat kidney cancer.

Aveo said last week that it was halting the renal cell carcinoma program and laying off 140 employees, 62 percent of its workforce. The cutback is expected to save the biotech about $190 million, allowing it to move forward with other drug programs.

Aveo and Astellas will continue to test tivozanib—considered by many doctors to be promising—as a treatment for breast cancer and colorectal cancer. Aveo is also pursuing other cancer drug programs, testing medicines to treat solid tumors and lung cancer.

Aveo executives have scheduled a conference call with investors Tuesday to discuss the FDA’s response letter and last month’s meeting of the FDA advisory committee in more detail. In a conference call last week, Aveo chief executive Tuan Ha-Ngoc apologized to patients, employees, and shareholders for Aveo’s failure to bring its kidney cancer drug to market.