First MCT8 deficiency therapy approved
Clinical studies showed reductions in circulating thyroid hormone levels and changes in heart rate and systolic blood pressure.
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By
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Kathryn Wighton
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September 29, 2026
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Clinical Scorecard: First MCT8 deficiency therapy approved
At a Glance
| Category | Detail |
|---|
| Condition | Monocarboxylate transporter 8 deficiency (Allan-Herndon-Dudley syndrome) |
| Key Mechanisms | Faulty gene affecting MCT8 transporter, leading to insufficient thyroid hormone in the brain and excess in the bloodstream. |
| Target Population | Primarily male patients with MCT8 deficiency. |
| Care Setting | Outpatient and inpatient settings for management of thyroid hormone levels. |
Key Highlights
- Tiratricol (Emcitate) approved for treating peripheral thyrotoxicosis in MCT8 deficiency.
- Tiratricol can enter cells without relying on the dysfunctional MCT8 transporter.
- Clinical studies showed reduced circulating thyroid hormone levels and improvements in cardiovascular symptoms.
- Common adverse effects include diarrhea, vomiting, rash, and excessive sweating.
- Patients on other thyroid medications should consult healthcare providers before starting tiratricol.
Guideline-Based Recommendations
Diagnosis
- Diagnosis of MCT8 deficiency based on clinical symptoms and genetic testing.
Management
- Tiratricol is administered once daily as a liquid suspension or via feeding tube.
Monitoring & Follow-up
- Monitor thyroid hormone levels, heart rate, and blood pressure during treatment.
Risks
- Risk of adverse effects such as diarrhea, vomiting, rash, and excessive sweating.
Patient & Prescribing Data
Infants through adults diagnosed with MCT8 deficiency.
Tiratricol received multiple designations including Orphan Drug and Breakthrough Therapy.
Clinical Best Practices
- Consult healthcare providers before starting tiratricol if on other thyroid medications.
- Regularly monitor patients for adverse effects and therapeutic efficacy.
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