March 01, 2019
3 min read
Save

Teprotumumab for treatment of thyroid eye disease meets endpoints in phase 3 study

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Results from the phase 3 OPTIC study showed more patients treated with teprotumumab had a meaningful improvement in proptosis compared with those who received placebo.

Perspective from Roger A. Dailey, MD, FACS

The confirmatory trial evaluated teprotumumab for the treatment of active thyroid eye disease.

The primary endpoint of the study was met, with 82.9% of 41 teprotumumab-treated patients achieving a 2 mm or more reduction of proptosis compared with 9.5% of 42 placebo patients, a statically significant difference (P < .001), according to a press release from Horizon Pharma.

All secondary endpoints were met in the study. A greater percentage of teprotumumab-treated patients experienced a 2 point or greater reduction in clinical activity score and a 2 mm or greater reduction in proptosis from baseline, with no deterioration in clinical activity score or proptosis in the fellow eye, compared with the placebo group.

A statistically significant percentage of teprotumumab patients experienced a clinical activity score value of 0 or 1 at week 24 in the study eye and a change from baseline of at least one grade in diplopia compared with the placebo group.

The safety profile of teprotumumab in this study was similar to phase 2 results. The dropout rate was less than 5%, and the majority of treatment-emergent adverse events were mild to moderate.

Teprotumumab has breakthrough therapy, orphan drug and fast track designations from the FDA, and Horizon expects to submit a biologics license application to the FDA this year.