FDA AdComm Votes in Favor of BLA for Teprotumumab for Thyroid Eye Disease

The US Food and Drug Administration (FDA) Dermatologic and Ophthalmic Drugs Advisory Committee (DODAC) voted 12-0 in favor of teprotumumab for Horizon Therapeutics on Friday.

The company previously submitted a Biologics License Application for the fully human monoclonal antibody, IGF-1R inhibitor, as the first potential treatment of active thyroid eye disease this year.

The unaminous vote supports the pivotal data benefits outweighing potential risks, as part of the company's application to the FDA.

With a PDUFA date scheduled for March 8, 2020, the intravenous treatment could be a few months away from becoming the first FDA-approved treatment for thyroid eye disease.

"Today’s unanimously positive vote marks a significant step towards the first FDA-approved treatment for patients with TED, a vision-threatening autoimmune disease," Timothy Walbert, chairman, president and chief executive officer, Horizon, said in a statement. "We believe that teprotumumab has the potential to address a significant unmet need for these patients and we look forward to working with the FDA as it completes its review of our application."

The FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee Meeting convened earlier today to discuss BLA 761143, which was submitted with support from multiple clinical trials including the phase 3 OPTIC study.

The OPTIC study, which was a 24-week randomized, double-masked, placebo-controlled trial included adults between the ages of 18 and 80 years old, with less than 9 months since active thyroid eye disease onset with no prior treatment. Patients included in the study received 8 intravenous infusions every three weeks for 21 weeks. The first injection was 10 mg/kg followed by 20mg/kg for the remaining seven infusions.

At the end of the 24-week study period, 40 patients had completed treatment in the placebo group and 39 completed from the teprotumumab. The overall responder rate at week 25 in patients in the teprotumumab group was 78% compared to 7.1% in the placebo group(P <0.001)—representing a difference of 70.82% (95% CI, 55.89-85.75).

In regard to proptosis, 82.9% of patients receiving teprotumumab experienced a reduction of 2 mm or more versus 9.5% in the placebo group. Investigators noted the difference between groups was 73.45% (95% CI, 58.89-88.01) the number needed to treat was 1.36. Investigators noted all secondary endpoints of the study were also met(P≤0.001).

Raymond Douglas, MD, PhD, professor of ophthalmology and director of the Orbit and Thyroid Eye Disease Center at Cedars-Sinai, who presented on behalf of Horizon Therapeutics during the advisory committee meeting, recently spoke with MD Magazine regarding the potential of the therapy.