UPDATE: It's official: The FDA advisory panel on oncologic drugs on Thursday voted 5 to 2 against recommending approval of Novartis' panobinostat.
- Novartis developed panobinostat for use in combination therapy with bortezomib and dexamethasone for treatment of multiple myeloma in patients who have received at least one prior treatment.
- Results were not encouraging: Compared with standard of care (standard chemotherapy treatment of bortezomib and dexamethasone), there was no statistically significant difference in overall survival, nor was there a difference in the treatment arms in terms of time-to-treatment failure. In addition, there was an increased incidence of death that was not due to the underlying cancer (7% vs. 3.5%).
- Despite these data, panobinostat delays disease progression by 2 to 4 months and generates a 6% improvement in overall response rate (ORR); therefore, it is not clear whether or not panobinostat will be approved, rejected, or approved with qualifications. But things aren't looking good.
When testing a drug in clinical trials, the big question is: "Do the risks outweigh the benefits?" The answers aren't always clear. Top-line results can be misleading, as can statistics and comments about statistical significance.
A deeper dive into the data shows that 60.7% of panobinostat-treated patients responded to treatment, compared with 54.6% of placebo-treated patients. Although there is a 6% improvement in overall response rate, with a P value of 0.09, the results are not statistically significant. There were serious adverse events in 60% of panobinostat-treated patients, compared with 42% in the placebo-treated patients. The most serious side effect, thrombocytopenia, affected 67% of panobinostat-treated patients, compared with 31% of placebo-treated patients.
Based on all of the factors above, approval is not likely.