An FDA advisory committee has unanimously backed teprotumumab as the first nonsurgical treatment for thyroid eye disease, according to a press release by Horizon Therapeutics.
“I don’t say this about every disease, but I hate this disease,” James Chodosh, MD, MPH, D.G. Cogan professor of ophthalmology at Harvard Medical School and the committee chairperson, said before casting a vote in favor of approval, according to Healio. “It is a devastating problem for patients. This is a disease that we need to do something for. This is a bad disease, and it has a tremendous impact on people’s lives. Though the numbers were small, the data presented was quite remarkable for a clinical trial.”
The 12-0 vote comes after the drug showed robust phase 3 efficacy in improving proptosis: 82.9% of teprotumumab patients achieved a reduction of 2 mm or more in proptosis compared with 9.5% of placebo patients. Secondary endpoints, including reduced diplopia, improved quality of life and reductions in Clinical Activity Score, were also met.
In briefing documents submitted to the FDA, the company claims that most adverse events were mild to moderate, with few discontinuations. Concerning adverse events in the teprotumumab group included hyperglycemia, worsening of pre-existing inflammatory bowel disease and hearing impairment. Approximately one-quarter of patients reported experiencing muscle spasms.
The monoclonal antibody, which targets the insulin-like growth factor 1 receptor, has already received fast track, breakthrough therapy and orphan drug designations. While the FDA typically follows the recommendations of the advisory committee when considering a biologics license, it is not obligated to do so. A decision is expected by March 8, 2020.