N-of-8 case study: Exanta (ximlegagatran) for anticoagulation

I was thinking back to the big ideas that often emerge from N-of-8 story development.

Many of these need more rigorous study. Of course, a great brand story must be supported by the science and must navigate treacherous regulatory waters.

One agent, Exanta (ximelagatran) looked quite promising as a new anticoagulation agent with less need for monitoring than the standard treatment, warfarin.   

This story seemed to be upheld in progressing clinical studies, right up until the FDA reviewed the submission for Exanta and rejected it. 

According to reports, the FDA did not approve ximelagatran primarily for safety reasons related to the drug's effect on liver function in 6% - 12% of patients taking ximelagatran for at least 3 to 6 months. 

However, the FDA failed to recognize the efficacy of ximelagatran, which was supported by the literature and was uniformly recognized by experts in the field.  Plus, its position on this specific agent also erected almost insurmountable hurdles for new drug development in the entire field.

Until recently.

It's very interesting to watch the novel oral anticoagulants enter the market with this backdrop.