Anyone open it? Page 232 Calculation of... Safety Margins "It is typical...by comparing group mean plasma concentrations....However plasma kinetics were not measured in some of the nonclinical studies conducted....or not measured at all dose levels. We have therefore estimated most safety margins.....#$%$? Did they forget? Page 204, in the pivotal trial....34 patients treated, 28 complete 26 weeks,23 enroll in OLE and only 5 complete the extension study. #$%$? This is a LIFE THREATENING DISEASE, with no current truly effective treatment.RIGHT? Why did the FDA give it a standard 10 month review.and not a priority review? That panel of experts did a wonderful job of ...........what?
page 198 primary efficacy results "Conclusion.......efficacy results are highly significant...over 26 weeks..." BUT "There are NO DATA(my caps) to show the efficacy of the drug for the possible long term treatment." As in over 26 weeks. Heck, they may approve it anyway .. The FDA did accept the NDA.... but this disease is caused by a genetic defect and doesn't just go away after 26 weeks. Just sayin'