Drugs / Tiratricol
Tiratricol
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | Emcitate | MCT8 deficiency | 2026-03-27 | “the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency” egetis.com ↗ |
| Filed | US (FDA) | Emcitate | MCT8 deficiency | 2026-01-29 | “Egetis has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency” egetis.com ↗ “completed the rolling New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) for Emcitate® (tiratricol)” egetis.com ↗ |
| Approved | EU (EMA) | Emcitate | — | 2025-02-12 | europa.eu ↗ |
| Filed | EU (EMA) | Emcitate | MCT8 deficiency | 2023-10-09 | “has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for Emcitate (tiratricol) for the treatment of MCT8 deficiency.” egetis.com ↗ “Submitted marketing authorisation application for Emcitate® (tiratricol) for the treatment of MCT8 deficiency to the European Medicines Agency EMA” egetis.com ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03783988 | Jan → Jun 2019 | skin atrophy | Trophea Development AB | Unknown | No outcome recorded |
| Phase 21 trial · 1 missed | ||||||
| Phase 2 | NCT02396459 | Dec 2020 → Jul 2027 expected | Allan-Herndon-Dudley syndrome | Rare Thyroid Therapeutics International AB | Active not recruiting | Missed primary |
| Phase 31 trial | ||||||
| Phase 3 | NCT05579327 | Jul 2023 → Jul 2025 | Allan-Herndon-Dudley syndrome, disorder of metabolite absorption and transport | Rare Thyroid Therapeutics International AB | Completed | Mixed |
| Phase not stated1 trial | ||||||
| — | NCT05911399 | — | Allan-Herndon-Dudley syndrome, disorder of metabolite absorption and transport | Rare Thyroid Therapeutics International AB | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 24
| Date | Issuer | Release |
|---|---|---|
| 2026-08-31 | Egetis Therapeutics | Regulatory Tiratricol Granted Orphan Drug Designation for MCT8 Deficiency in Japan egetis.com ↗ |
| 2026-04-08 | Egetis Therapeutics | Regulatory Egetis Receives Conditional FDA Acceptance of Proprietary Name Emcitate® egetis.com ↗
Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the U.S. Food and Drug Administration (FDA) has completed their review of the proposed proprietary name Emcitate and have concluded that it is conditionally acceptable. |
| 2026-03-27 | Egetis Therapeutics | Regulatory Egetis Announces FDA Acceptance and Priority Review of NDA for Emcitate® (tiratricol) for MCT8 Deficiency egetis.com ↗
Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency. |
| 2026-02-26 | Egetis Therapeutics | Regulatory Year-End Report January-December 2025 egetis.com ↗
Egetis submitted the U.S. NDA for Emcitate® (tiratricol) for treatment of MCT8 deficiency |
| 2026-01-29 | Egetis Therapeutics | Regulatory Egetis Completes the U.S. Rolling NDA Submission for Emcitate® (tiratricol) for Treatment of MCT8 Deficiency egetis.com ↗ |
| 2025-12-19 | Egetis Therapeutics | Regulatory Egetis Therapeutics initiates New Drug Application in the USA for Emcitate® (tiratricol) for MCT8 deficiency egetis.com ↗
Consequently, the Company has now submitted the non-clinical and quality (chemistry, manufacturing and controls, or CMC) sections of the NDA to the FDA. |
| 2025-12-04 | Egetis Therapeutics | Results New publication highlights meaningful clinical benefits of Emcitate® (tiratricol) in treating Resistance to Thyroid Hormone Beta egetis.com ↗ |
| 2025-11-14 | Egetis Therapeutics | Results Egetis announces positive results from the ReTRIACt study of Emcitate® (tiratricol) in MCT8 deficiency egetis.com ↗
Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), today announced positive topline results from the ReTRIACt study of Emcitate® (tiratricol) in patients with MCT8 deficiency. |
| 2025-10-23 | Egetis Therapeutics | Regulatory Egetis granted a rolling NDA review by FDA for Emcitate® (tiratricol) based on currently available clinical data egetis.com ↗
As agreed with the FDA, the rolling NDA submission will commence in December 2025, targeting a complete NDA submission in early 2026 and completion of the FDA review process in Q3 2026, if Priority Review is granted |
| 2025-07-15 | Egetis Therapeutics | Regulatory Egetis receives FDA Breakthrough Therapy Designation for tiratricol for MCT8 deficiency egetis.com ↗
Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tiratricol, as a treatment for monocarboxylate transporter 8 (MCT8) deficiency. |
| 2025-04-25 | Egetis Therapeutics | Results Article mapping variants in thyroid hormone transporter MCT8 to disease severity published in Nature Communications egetis.com ↗
treatment with tiratricol (Emcitate®) was equally effective in reducing serum total T3 concentrations, as well as improving clinical and biochemical outcomes irrespective of the functional impact of the underlying MCT8 variant |
| 2025-02-13 | Egetis Therapeutics | Regulatory European Commission approves Egetis’ Emcitate® (tiratricol) as the first and only treatment for patients with MCT8 deficiency egetis.com ↗
Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), today announced that the European Commission (EC) has approved Emcitate® (tiratricol) for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency. |
| 2024-12-12 | Egetis Therapeutics | Regulatory Egetis receives positive CHMP opinion for Emcitate® (tiratricol) for the treatment of MCT8 deficiency egetis.com ↗
Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Emcitate® (tiratricol) for “The treatment of MCT8 deficiency”. |
| 2024-08-28 | Egetis Therapeutics | Results New post-hoc analysis reports effects of tiratricol on patient-centered outcome measures in patients with MCT8 deficiency egetis.com ↗
According to the Abstract, there were improvements upon tiratricol treatment reported by caregivers related to improved interaction (22/39), greater alertness (19/39), improved motor skills (12/39), improved head control (7/39), and improved sleep (8/39). |
| 2024-08-21 | Egetis Therapeutics | Results New data shows tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with survival benefits egetis.com ↗
In the Abstract, treatment with the investigational drug tiratricol (Emcitate®) in paediatric and adult patients with MCT8 deficiency is associated with an approximately three times lower risk of mortality compared to MCT8 deficiency patients not treated with tiratricol. |
| 2024-08-14 | Egetis Therapeutics | Regulatory Egetis submits responses to the European Medicines Agency’s Day 120 List of Questions for the Marketing Authorisation Application for tiratricol (Emcitate®) egetis.com ↗
Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), today announced that the Company has submitted responses to the Day 120 List of Questions received from the European Medicines Agency for the Marketing Authorisation Application (MAA) for tiratricol (Emcitate®) for the treatment of MCT8 deficiency. |
| 2024-06-19 | Egetis Therapeutics | Results Egetis announces topline results of the Phase 2 Triac Trial II with Emcitate® (tiratricol) for MCT8 deficiency egetis.com ↗
The numerical improvements versus baseline observed on the primary endpoints of neurocognitive development assessed by the GMFM-88 and BSID-III scales did not show a statistically significant improvement versus historical controls. |
| 2024-06-03 | Egetis Therapeutics | Regulatory Egetis awarded Promising Innovative Medicine designation in the UK for Emcitate® (tiratricol) for treatment of MCT8 deficiency egetis.com ↗
A Promising Innovative Medicine (PIM) designation is a recognition of Emcitate® (tiratricol) as a promising candidate drug in the UK for the treatment of patients with MCT8 deficiency. |
| 2023-10-27 | Egetis Therapeutics | Regulatory Egetis announces EMA validation of Marketing Authorisation Application for Emcitate for the treatment of MCT8 deficiency egetis.com ↗ |
| 2023-10-09 | Egetis Therapeutics | Regulatory Egetis submits marketing authorisation application for Emcitate for treatment of MCT8 deficiency to the European Medicines Agency egetis.com ↗
Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), today announced that the Company has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for Emcitate (tiratricol) for the treatment of MCT8 deficiency. |
| 2022-12-07 | Egetis Therapeutics | Regulatory Egetis submits Expanded Access Program for Emcitate® in the USA egetis.com ↗
Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) today announced that the Company has submitted a protocol for an Expanded Access Program for Emcitate (tiratricol) in the USA, as requested by the Food and Drug Administration (FDA). |
| 2022-03-24 | Egetis Therapeutics | Regulatory Egetis receives positive EMA opinion for orphan designation of Emcitate for treatment of Resistance to Thyroid Hormone type beta (RTH-β) egetis.com ↗
The positive opinion adopted by the EMA on the Orphan Medicinal Product submission for RTH-β follows the Orphan Drug Designation for the treatment of RTH-β recently granted by the US Food and Drug Administration (FDA). |
| 2022-01-18 | Egetis Therapeutics | Regulatory Egetis concludes that demonstrating treatment effects on T3 levels in MCT8-deficiency could provide a basis for marketing approval for Emcitate® in the US egetis.com ↗
In recent positive regulatory interactions, FDA acknowledges that a treatment effect on T3 levels and the manifestations of chronic thyrotoxicosis in MCT8-deficiency could provide a basis for marketing approval also in the US. |
| 2021-10-25 | Egetis Therapeutics | Results New data confirms long-term efficacy and safety of Emcitate® in MCT8 deficiency patients egetis.com ↗
The primary endpoint, the mean serum T3 concentrations, decreased significantly from 4.58 (SD: 1.11) nmol/L at baseline, to 1.66 (0.69) nmol/L at last visit; target: 1.4–2.5 nmol/L (mean decrease 2.92 nmol/L, 95% CI 2.61-3.23 nmol/L, p<0.0001). |
All press releases naming this drug 33 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Tiratricol | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05579327 ↗ |
| Known as | 3,3',5-triiodothyroacetic acid | “Tiratricol (3,3',5-triiodothyroacetic acid) is available as 350 µg tablets for oral administration (suspended in water and, if needed, mixed with food) or by percutaneous...” NCT05911399 ↗ |
| Known as | TRIAC | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03783988 ↗ |
| Known as | triiodothyroacetic acid | “The new topical gel contains dehydroepiandrosterone (DHEA) and triiodothyroacetic acid (TRIAC)” NCT03783988 ↗ |
| Route | Oral | “administered orally” NCT05579327 ↗ |