Genentech receives FDA Refuse to File Letter for T-DM1

[SunDiego]

This makes me sick.

http://www.google.com/hostednews/ap/...fBFFwD9HRUKQG0

Here is the key sentence in the release: "In its review of the BLA, the FDA stated the T-DM1 trials did not meet the standard for accelerated approval because all available treatment choices approved for metastatic breast cancer, regardless of HER2 status, had not been exhausted in the study population."

In other words, Genentech made a mistake in which clinical trials it chose to do. The current study can't serve as the basis for approval. The company says it "believes firmly" in T-DM1, and that it plans to file for approval in mid-2012 based on as-yet-uncompleted studies.

Essentially this delays by at least TWO YEARS the FDA approval of TDM-1. What was Immunogen thinking?! This doesn't change the trials, but certainly a major setback for those (like my wife) who was hoping access it outside of a trial.

[sarah]

This is terrible and the fact that they say "the T-DM1 trials did not meet the standard for accelerated approval because all available treatment choices approved for metastatic breast cancer, regardless of HER2 status, had not been exhausted in the study population."
what has non-HER2 bc got to do with HER2 positive cancer????!!!
It's outrageous and a letter writing campaign to representatives in Congress is in order. I'm sure Genetech will be helpful in suggesting the best course of action.
sarah

[Hopeful]

FDA Guidance on "Refuse to File":http://www.fda.gov/downloads/Drugs/G.../UCM080561.pdf

Hopeful

Or, it could mean that Genentech was given faulty guidance on the early application and then the FDA backtracked. It could also mean that the FDA didn't like how Avastin played out and is now making sure they don't approve a drug and need to backtrack on it so it sounds like it could be a purely political move by the FDA. Wouldn't be the first time. I hope the breast cancer groups scream loudly on this one, they need to on behalf of the patients. Unfortunately, the way oncology clinical trials are designed, based on FDA guidance and regulations, death is the primary endpoint with the better, more effective drugs often taking longer to approve because they WORK. Talk about bitter irony!