FDA approves tiratricol as first treatment for MCT8 deficiency

The FDA approved tiratricol, sold as Emcitate, to treat peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. The rare genetic disorder primarily affects males. The FDA said tiratricol can enter cells without relying on the faulty MCT8 transporter, and is the first FDA-approved treatment for the condition.

Approval was supported by two studies, according to MedPage Today: a randomized, placebo-controlled trial with 20 participants ages 5 to 31 years, and an open-label study with 46 participants ages 10 months to 66.8 years. Across the studies, tiratricol reduced excess thyroid hormone levels in the blood and improved cardiovascular and metabolic symptoms, including systolic blood pressure and heart rate. The drug is taken once daily by mouth or through a feeding tube. The most common side effects were diarrhea, vomiting, rash and excessive sweating. Egetis Therapeutics said it expects to make the drug available in the U.S. within 8 to 10 weeks.