Pipeline Overview
Candidate
EMCITATE® (tiratricol) EU
MCT8 deficiency
MAA/NDA
- EC approval received Feb 12, 2025
- Launched in Germany May 1, 2025
EMCITATE® (tiratricol) U.S.
MCT8 deficiency
MAA/NDA
- NDA accepted Mar 27, 2026
- Priority Review granted
- FDA approved Sep 28, 2026
tiratricol
RTH-beta*
Preclinical
- ODD granted by FDA & EMA in 2022
- Finalizing development plan
EC: European Commission; EMA: European Medicines Agency; MAA: Marketing Authorization Application (EU); MCT8: Monocarboxylate Transporter 8; NDA: New Drug Application (USA); ODD: Orphan Drug Designation; RTH-beta: Resistance to Thyroid Hormone beta.
*tiratricol in RTH-beta is an investigational product and has not been approved for use in any market
Resistance to Thyroid Hormone beta (RTH-beta)
Resistance to Thyroid Hormone beta (RTH-beta) is a rare endocrine disorder caused by mutations in the thyroid hormone receptor beta (THRB) gene
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