Raymond Douglas, MD, PhD: Teprotumumab for Thyroid Eye Disease Provides Long-Term Benefit

Teprotumumab (TEPEZZA) is associated with long-term clinical improvements for patients with thyroid eye disease, according to new data presented at the American Academy of Ophthalmology (AAO) 2020 conference. Notably, these findings demonstrated improvements in the less severe eye—or fellow eye.

Currently, teprotumumab is the first and only medicine approved by the U.S. Food and Drug Administration (FDA) for the treatment of thyroid eye disease.

The findings, presented by Raymond Douglas MD, PhD, Director of Orbital and Thyroid Eye Disease Program, included additional data from the OPTIC Phase 3 clinical trial and OPTIC-X open-label extension trial.

In the OPTIC 48-week off follow-up trial, the majority of patients (11/19; 58%) treated with teprotumumab who had ≥1 grade of diplopia improvement at Week 24 in OPTIC maintained their response at Week 72 without additional thyroid eye disease treatment.

Furthermore, 50% of teprotumumab-treated patients who had a diplopia score of 0 at Week 24 maintained the same score at Week 72 without additional treatment.

The OPTIC-X findings showed that 61% of patients who had initially received placebo during the OPTIC trial were considered diplopia responders (≥1 grade improvement) at Week 24 with teprotumumab.

The study also noted that 5 initially treated placebo patients during the OPTIC trial achieved a CAS of 0 or 1, indicating minimal or no inflammation. Of those patients, 60% responded to proptosis at Week 24.

A pooled analysis of the TEPEZZA Phase 2 and 3 trials demonstrated that the drug may offer benefits in patients with less severe thyroid eye disease.

Investigators analyzed the intention-to-treat population from both trials—in total, these patients (n=171) were randomized 1:1 to either the teprotumumab or placebo cohort.

Overall, they found that more teprotumumab-treated patients experienced an improvement in proptosis (≥2 mm reduction) in the fellow eye than placebo patients (63.1% vs. 8.0%; P<.001).

Furthermore, the mean proptosis reduction for teprotumumab patients was 2.39 mm—versus a 0.15 mm increase for placebo patients.

“What every ophthalmologist needs to know is that there is this new dawn arising—that there is a new day in this field—where this is a medication that changes the course of this disease,” Douglas said in an interview with HCPLive®.

“The key takeaway is there’s a new whole new landscape,” he concluded.