Upneeq

Approval was based on data from 2 double-masked, vehicle-controlled, parallel-group phase 3 trials (Trial 1 and Trial 2) that evaluated the efficacy and safety of Upneeq in 304 patients with acquired blepharoptosis. Patients were randomly assigned 2:1 to receive Upneeq once daily or vehicle for 42 days. 

The primary endpoint was the mean change from baseline in the number of points seen on the Leicester Peripheral Field Test in the study eye at hour 6 on day 1 and hour 2 on day 14. A secondary end point included the change from baseline in marginal reflex distance 1 (MRD1).

In both studies, improvement in superior visual field was observed with Upneeq at the 2-hour time point and maintained at the 6-hour time point. Results from Trial 1 (N=140) showed that Upneeq met the primary endpoint demonstrating a statistically significant improvement at hour 6 on day 1 (mean difference 3.7; 95% CI, 1.8-5.6; P <.01) and at hour 2 on day 14 (mean difference 4.2; 95% CI, 2.0-6.0; P <.01) vs vehicle. 

Additionally, statistically significant improvements in the Upneeq treatment arm were noted in Trial 2 (N=164) at hour 6 on day 1 (mean difference 4.2; 95% CI, 2.4-6.1; P <.01) and at hour 2 on day 14 (mean difference 5.3; 95% CI, 3.7-7.1; P <.01) vs vehicle. Both studies demonstrated greater MRD1 increases at both time points vs vehicle.