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. Viridian Therapeutics, the manufacturer, announced plans for an immediate launch of the drug.

Veligrotug is indicated for both 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 of veligrotug was based on the results of two phase III trials, known as THRIVE and THRIVE-2, which met their primary and all secondary endpoints. In the THRIVE trial, patients with active disease receiving veligrotug showed an average proptosis reduction of 2.89 mm by week 15, compared to a 0.48 mm reduction in the placebo group. For patients with chronic disease in the THRIVE-2 trial, veligrotug-treated participants experienced proptosis reductions of 2.34 mm, while the placebo group saw reductions of 0.46 mm.

The trials also evaluated diplopia, or double vision. Complete diplopia resolution occurred in 54% of veligrotug-treated participants with active disease, compared to 12% in the placebo group. In participants with chronic disease, 32% of those treated with veligrotug achieved complete diplopia resolution, whereas 14% of the placebo group did. A reduction of at least 1 point on the Gorman subjective diplopia scale, defined as a diplopia response, was observed in 63% of veligrotug-treated participants with active disease versus 20% of the placebo group. For chronic disease patients, a diplopia response occurred in 56% of veligrotug-treated participants compared to 25% for placebo.

Michael Yen, an MD and THRIVE investigator at Baylor College of Medicine, noted the drug's effectiveness. "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," Yen said. "This is an exciting new option for physicians to offer their TED patients."

Veligrotug is administered via five intravenous infusions over a 12-week period, with each infusion given once every three weeks. The drug's label states there are no contraindications. However, it lists warnings for potential infusion reactions, inflammatory bowel disease, hyperglycemia, and hearing impairment. Common adverse events, with an incidence of 5% or more, included muscle spasms, headache, hearing impairment, hyperglycemia, fatigue, diarrhea, ear discomfort, infusion-related reaction, nausea, nasopharyngitis, increased blood creatine phosphokinase, dry skin, and hypertension.

Viridian Therapeutics has established a patient support program to assist with insurance navigation and out-of-pocket costs for veligrotug. The approval introduces a new non-surgical treatment for thyroid eye disease, expanding options beyond teprotumumab (Tepezza), which was previously the only approved non-surgical therapy targeting the insulin-like growth factor 1 receptor (IGF-1R). Teprotumumab targets the IGF-1R. The immediate launch by Viridian Therapeutics aims to make the treatment available to patients and healthcare providers.