- Emcitate (tiratricol) is the first FDA-approved therapy for MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome, a rare genetic disorder that primarily affects males.
- Tiratricol bypasses the broken MCT8 transporter by entering cells on its own, reducing elevated blood thyroid hormone levels and improving cardiovascular and metabolic symptoms.
- Effectiveness was evaluated in an international, multi-center, randomized, placebo-controlled trial (NCT05579327) and a longer-term open-label study spanning infants to adults.
- The approval was granted to Egetis Therapeutics US Inc., with Orphan Drug, Rare Pediatric Disease, Fast Track, Breakthrough Therapy designations and Priority Review.
Quotes
“Until now, patients living with MCT8 deficiency and their families had no FDA-approved treatment option”
“This drug sidesteps that problem, as its active ingredient, tiratricol, can enter cells on its own without relying on the broken transporter, leading to a decrease in the elevated blood thyroid hormone levels.”