Cell Therapeutics Lymphoma Drug Fails to Win FDA Approval

4/9/10Follow @xconomy

Cell Therapeutics suffered what I called a “humiliating public beatdown” last month in front of an FDA advisory committee, and now the FDA has made it official in writing. The Seattle-based biotech company said its application to market a new lymphoma drug in the U.S. has been formally turned down by the FDA.

The FDA apparently didn’t need to wait until its statutory deadline of April 23 to deliver the bad news in its “complete response” letter to the company. The agency said that if Cell Therapeutics wants clearance to start marketing pixantrone (Pixuvri) in the U.S. for patients with non-Hodgkin’s lymphoma, it will need to run another clinical trial to prove it is safe and effective. Cell Therapeutics said it has had some preliminary talks with the FDA about what such a trial would look like, and that it plans to pursue an “expanded access” program to make pixantrone more widely available to patients even without being able to sell it.

The FDA’s rejection is no surprise, given the harsh public comments made last month at a panel of the Oncologic Drugs Advisory Committee. The panel voted 9-0 against recommending approval of pixantrone. The FDA’s chief cancer drug reviewer, Richard Pazdur, slammed the application in his remarks, noting that Cell Therapeutics was seeking approval on the basis of a “single, incomplete trial.” The chair of the FDA panel, Gail Eckhardt of the University of Colorado at Denver, said the Cell Therapeutics application was “disturbing,” partly because it only enrolled 140 of the 320 patients needed to generate a statistically valid result the way the trial was originally designed.

Cell Therapeutics took the unusual step of quoting a cancer researcher in its press release who took a critical shot at the FDA.

“This is a sad outcome for our patients with relapsed/refractory aggressive NHL,” said Dr. Stanley Marks, chief medical officer for the University of Pittsburgh Cancer Centers, in a Cell Therapeutics statement. “I was disappointed that an agency charged with providing treatment hope for patients with life threatening diseases like relapsed/refractory non-Hodgkin’s lymphoma would ignore clinically meaningful improvements in overall response rate and progression-free survival, let alone complete responses, something we all wish for our patients, but with existing treatments rarely achieve.”

Cell Therapeutics, as I noted in an analysis after the FDA panel, still says it hopes it can win approval in Europe. The company is planning to submit that application in the three-month period that ends in September.

Getting an approval in the U.S. is a remote possibility, according to David Miller, an analyst with Biotech Stock Research in Seattle. A new trial would require 300 or more patients, many of them in the U.S., to address some of the FDA panel’s prior concerns, and it will take years to enroll them all, Miller said in a note to clients. The trial would cost $30 million to $50 million, which isn’t something the company can afford with its existing cash reserves.

Still, Cell Therapeutics has raised $1.4 billion since its founding in 1991, and CEO James Bianco was able to lead a $20 million fundraising from investors even after the FDA panel’s brutal dismissal of the company’s drug. While the company said it sees reasons for hope with its “expanded access” program to offer the drug in the U.S. to patients while it is being studied, plus the chance of a European approval, Miller dismissed those possibilities.

“CTI can keep preying on novice investors with stories about EU approval and Expanded Access. At some point, however, we think they will run out of novices and the market cap will return to levels appropriate with their cash and debt situation,” Miller wrote.

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