Phase III results for Esmya (ulipristal acetate) demonstrates management of uterine fibroids- Gedeon Richter

Gedeon Richter announced results of PEARL IV, a Phase III, randomized, double-blind, parallel group study investigating the efficacy and safety of repeated 3 months treatment courses of Esmya (ulipristal acetate or UPA) daily 5mg or 10mg for the long-term management of symptomatic uterine fibroids, characterized by heavy bleeding. The study, which investigated 4 treatment courses in patients across 46 European centres in 11 countries, showed approximately 93% of women or greater achieved controlled bleeding at the end of each treatment course and at least 76% of women were in amenorrhoea at the end of each treatment courses. There was also a 67% median reduction in fibroid volume at the end of treatment course 4.

Additionally, 65.5%, 73.5%, 74.7% and 78.1% of patients had clinically significant reduction of the three largest fibroids (i.e. ≥25% reduction) after each consecutive treatment course with UPA 5mg. Finally, 73.5% of women experienced both amenorrhea and clinically significant fibroid reduction (≥25%) with 4 repeated courses. The use of ulipristal acetate showed an improvement in quality of life (QoL) and pain which was maintained during the off treatment intervals, and the safety profile for UPA in this trial confirmed the previously reported safety profile. The vast majority of AEs (97.6%) were of mild or moderate severity in both groups. Hot flushes and headaches were the most frequently reported adverse reactions.

Comment: The initial EU marketing authorisation for UPA 5mg was granted in 2012 for the pre-operative treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. The new indication, allowing intermittent medical treatment of the moderate to severe symptoms of uterine fibroids with UPA 5mg, was granted in May 2015 and provides an important opportunity for many women suffering from this condition to potentially avoid surgical interventions.