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

Veligrotug is the first treatment labeled for both active and chronic forms of thyroid eye disease. The approval was based on two phase III trials, THRIVE and THRIVE-2, which met their primary and all secondary endpoints.

In the THRIVE trial, patients with active disease who received veligrotug experienced an average proptosis reduction of 2.89 mm by week 15, compared to 0.48 mm in the placebo group. The THRIVE-2 trial, involving patients with chronic disease, showed proptosis reductions of 2.34 mm with veligrotug, versus 0.46 mm with placebo.

Trials demonstrated an effect on diplopia response and complete resolution in both active and chronic forms of the disease. Complete diplopia resolution occurred in 54% of veligrotug-treated participants with active disease, compared with 12% in the placebo group. For chronic disease, complete diplopia resolution was observed in 32% of veligrotug-treated participants, compared with 14% of those receiving 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." Veligrotug functions as a full antagonist humanized monoclonal antibody targeting the insulin-like growth factor 1 receptor (IGF-1R).

The label for veligrotug includes warnings for potential adverse events such as infusion reactions, inflammatory bowel disease, hyperglycemia, and hearing impairment. Common adverse events, occurring in 5% or more of participants, 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, totaling a 12-week treatment course. Viridian Therapeutics plans an immediate launch of veligrotug and has established a patient support program to assist with insurance and out-of-pocket costs.