Biotech Values

[jq1234]

I didn't see their trial being stopped early for efficacy.

Have you ever seen a SINGLE ARM trial stopped for efficacy applying for regulatory approval? I haven't.

I can't say I understand the FDA's reluctance in their case and it certainly does give pause to anyone looking for accelerated approval for a condition with lots of existing alternative treatments.

Not necessarily FDA. FDA conducted advisory panel on accelerated approval on Feb 8. Majority Advisory panel members said that companies seeking accelerated approval for cancer drugs should submit at least one controlled trial to bolster their application. Although the question wasn’t a voting question, it was apparent that a majority of the Panel felt that the bar set by FDA currently was too low.

I personally don't think FDA will issue uniform decision regardless of cancer indications. Most likely accelerated approval bar will be raised to randomized trial with surrogate endpoint, or single arm in truely refractory patients without any alternative.