U.S. — The U.S. Food and Drug Administration (FDA) approved veligrotug (Lumvoa) for the treatment of thyroid eye disease regardless of disease activity or duration.

Veligrotug is the first treatment with labeling for both the active and chronic forms of thyroid eye disease. The drug functions as a full antagonist humanized monoclonal antibody targeting the insulin-like growth factor 1 receptor (IGF-1R).

The approval was based on results from two phase III trials that met their primary and all secondary endpoints. In the THRIVE trial, patients with active disease who received veligrotug achieved an average proptosis reduction of 2.89 mm by week 15, compared to 0.48 mm in those given a placebo. In the THRIVE-2 trial, patients with chronic disease achieved proptosis reductions of 2.34 mm with veligrotug and 0.46 mm with placebo.

Complete diplopia resolution occurred in 54% of veligrotug-treated participants with active disease, compared with 12% of those receiving placebo. In chronic disease patients, 32% of veligrotug-treated participants had complete diplopia resolution, compared with 14% in the placebo group. Diplopia response occurred in 63% of veligrotug-treated participants with active disease versus 20% for placebo, and 56% for veligrotug-treated participants with chronic disease versus 25% for placebo.

Michael Yen, MD, a THRIVE investigator at Baylor College of Medicine, said, "It's encouraging to see a new treatment for the full spectrum of the disease with data showing rapid onset of proptosis reduction as well as improvements in diplopia." He added, "This is an exciting new option for physicians to offer their TED patients."

There are no contraindications to veligrotug. The drug's label includes warnings related to infusion reactions, inflammatory bowel disease, hyperglycemia, and hearing impairment. The most common adverse events, occurring in 5% or more of patients, included muscle spasms, headache, hearing impairment, hyperglycemia, fatigue, diarrhea, ear discomfort, infusion-related reaction, nausea, nasopharyngitis, increased blood creatine phosphokinase, dry skin, and hypertension. Veligrotug is administered via five intravenous infusions given once every three weeks over a 12-week period.

Before veligrotug's approval, teprotumumab (Tepezza) was the only non-surgical treatment available on the market for thyroid eye disease. Viridian Therapeutics plans to launch veligrotug immediately and has developed a patient support program to assist with insurance and out-of-pocket costs. The company is also conducting phase III studies for another IGF-1R monoclonal antibody, elegrobar, for thyroid eye disease.