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First MCT8 deficiency therapy approved

Clinical studies showed reductions in circulating thyroid hormone levels and changes in heart rate and systolic blood pressure.

  • By

  • Kathryn Wighton

  • September 29, 2026

  • 3 min

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  • 1

    The FDA approved tiratricol (Emcitate) for treating peripheral thyrotoxicosis in patients with MCT8 deficiency.

  • 2

    MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome, primarily affects males and is caused by a faulty gene.

  • 3

    Tiratricol can enter cells independently of the MCT8 transporter, reducing elevated thyroid hormone levels in the blood.

  • 4

    Two clinical studies demonstrated tiratricol's efficacy in lowering thyroid hormone levels and improving cardiovascular symptoms.

  • 5

    Common adverse effects of tiratricol include diarrhea, vomiting, rash, and excessive sweating, with specific medication interactions noted.

Original Source(s)

First MCT8 deficiency therapy approved

  • by Kathryn Wighton

  • September 29, 2026

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