Maybe now people will start to take a hard look at the other RPRX data. The LFTs was something that had to reveal. I am sure they delayed this information as long as possible. In the PR, RPRX makes claims about significant improvements in the “quality of life parameters.” They carefully do NOT state when these p values are obtained. Recall, the duration of treatment is four (4) months.
Completed studies at 25 and 12.5 mg doses have demonstrated statistically and clinically significant control of excessive menstrual bleeding and improvement in quality of life parameters. In a completed Phase II study, which included 127 women with uterine fibroids, doses of 25 and 12.5 mg were compared to placebo for a period of three months. Both the 25 and 12.5 mg doses showed statistically significant (p < 0.0001) improvements in three clinically relevant endpoints: hemoglobin level, menstrual bleeding scores and quality of life parameters.
What they do not state, these improvements were only found after three (3) months, not before. They discontinue the drug after month four (4). At most, the drug is helpful 50% of the time. Why do they discontinue the drug? The reason is unscheduled bleeding and emergency procedures, much more than LFT changes.
Also, the percent of women pain free is less than 50% for both 12.5 and 25 mg. Moreover, for the 12.5 mg treatment arm the percent is less than 30%.
Assuming no LFT changes, no unscheduled bleeding, and no emergency procedures, will the FDA approve a drug that is effective at most 50% of the time. After stopping the drug, there is the known concern for bleeding in the off-drug interval. Now, add back the known problems for unscheduled bleeding and emergency procedures, what is the chance for approval?
AI
