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Emcitate (tiratricol)

MCT8 deficiency

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78 sources

Company-reported prevalence

Emcitate

Overview

FieldValue
DrugEmcitate (tiratricol)
IndicationMCT8 deficiency
DiseaseMonocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley syndrome)
CompanyEgetis Therapeutics AB, through its subsidiary Rare Thyroid Therapeutics International AB (marketing authorization holder in the EU; own commercialization in the United States and Europe); Fujimoto Pharmaceutical Corporation (exclusive development and commercialization license, Japan); Er-Kim (exclusive distribution, Turkey and further markets in central, eastern, and southeastern Europe); taiba rare (exclusive distribution and early access, Saudi Arabia, United Arab Emirates, Qatar, Oman, and Bahrain)
Documents searchedWebsite pages for healthcare professionals (MCT8 deficiency; Emcitate), for patients and caregivers (what is MCT8 deficiency; educational resources), pipeline, and investors (accessed October 2026); disease awareness sites mct8deficiency.com and lifewithmct8deficiency.com (accessed October 2026); emcitate.com (accessed October 2026); press releases and interim reports published on egetis.com, January 2023 to October 2026 (including November 10, 2023, December 13, 2023, December 19, 2023, January 29, 2026, March 27, 2026, and September 28, 2026); interim reports for the first quarter of 2025 (April 30, 2025), the fourth quarter of 2025 (February 26, 2026), the first quarter of 2026 (April 29, 2026), and the second quarter of 2026 (August 21, 2026); annual report for 2025 (Swedish, March 24, 2026); second quarter 2026 webcast presentation (August 21, 2026); benefit assessment dossier Module 3 A submitted to the German Federal Joint Committee (April 29, 2025)
Data as ofOctober 14, 2026
Prepared byAI research agent (Claude); not yet reviewed by a person

Company statements of prevalence

United States

Number treated: Egetis Therapeutics interim report for the second quarter of 2026, August 2026

“At FDA’s request, Egetis has implemented an Expanded Access Program (EAP) in the U.S. Currently, 17 hospitals are included and approximately 60 patients are receiving tiratricol in the EAP.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2026) interim report for the second quarter of 2026, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Number treated: Egetis Therapeutics second quarter 2026 webcast presentation, August 2026

“~60 patients across 17 sites in EAP today” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2026) second quarter 2026 webcast presentation, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 7 of 13 (opens in a new tab)

Number treated: Egetis Therapeutics interim report for the first quarter of 2026, April 2026

“Currently approximately 50 patients are receiving tiratricol in the EAP.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2026) interim report for the first quarter of 2026, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Number diagnosed: Egetis Therapeutics press release, December 2023

“This has resulted in the identification of an additional 50 MCT8 deficiency patients in the USA, previously not diagnosed or known to the Company.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2023) press release, December 13, 2023, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Europe

Prevalence: Rare Thyroid Therapeutics International benefit assessment dossier for Germany, April 2025

“Zusammenfassend ergibt sich eine Prävalenz zwischen 1:1.890.000 und 1:1.000.000.” (opens the source at this quote in a new tab)

“So ergibt sich eine theoretische neue Obergrenze von 1:666.666 für Deutschland.” (opens the source at this quote in a new tab)

Cited source: 2.1 European Medicines Agency 2018; 2.2 Kubota 2022; 2.3 Egetis Therapeutics 2025

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 26 of 67 (opens in a new tab)

Number affected: Rare Thyroid Therapeutics International benefit assessment dossier for Germany, April 2025

“Spanne von 45-127 Patienten in Deutschland im vorliegenden Anwendungsgebiet” (opens the source at this quote in a new tab)

Cited source: 2.3 Egetis Therapeutics 2025; 2.4 Statistisches Bundesamt 2024

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 27 of 67 (opens in a new tab)

Eligible population: Rare Thyroid Therapeutics International benefit assessment dossier for Germany, April 2025

“Wird dieser Anteil nun auf die geschätzte Zahl der MCT8-Patienten übertragen ergibt sich ein Mittelwert von 71 in einem Schwankungsbereich von 37-106.” (opens the source at this quote in a new tab)

Cited source: 2.1 European Medicines Agency 2018; 2.2 Kubota 2022; 2.3 Egetis Therapeutics 2025

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 29 of 67 (opens in a new tab)

Global

Number treated: Egetis Therapeutics press release, November 2023

“around 190 patients in over 25 countries around the world, are already being treated with Emcitate as part of Egetis’ Managed Access programs.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2023) press release, November 10, 2023, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Other countries and regions

Number treated: Egetis Therapeutics annual report for 2025, March 2026

“Emcitate har förskrivits via ’Managed Access Program’ (MAP) till över 230 patienter efter godkännande av nationella” (opens the source at this quote in a new tab)

“i över 25 länder, innan godkännande av Emcitate i EU 2025.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2026) annual report for 2025, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 55 of 103 (opens in a new tab)

Number affected: Egetis Therapeutics press release, November 2023

“Kubota et al. (Ref. 2) in turn have conducted a nationwide survey of MCT8 deficiency and estimated there are over 60 cases with the disease in Japan” (opens the source at this quote in a new tab)

Cited source: 2.2 Kubota 2022

Egetis Therapeutics (2023) press release, November 10, 2023, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Number diagnosed: Egetis Therapeutics press release, November 2023

“Iwayama et al. (Ref. 1) have reviewed the literature of MCT8 deficiency in Japan, and identified 36 already published cases.” (opens the source at this quote in a new tab)

Cited source: 2.6 Iwayama 2021

Egetis Therapeutics (2023) press release, November 10, 2023, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Geography not stated

Prevalence: Egetis Therapeutics website, October 2026

“Epidemiologic data for MCT8 deficiency are limited but suggest an overall population prevalence of fewer than 1 to 3 per million.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2026) website, MCT8 deficiency page for healthcare professionals, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Incidence: Egetis Therapeutics press release, January 2026

“Estimated incidence is 1 in 70,000 males” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2026) press release, January 29, 2026, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Prevalence: Egetis Therapeutics annual report for 2025, March 2026

“drabbar tillståndet nästan enbart män, med en uppskattad frekvens på 1 av 70 000 män.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2026) annual report for 2025, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 12 of 103 (opens in a new tab)

Number treated: Egetis Therapeutics interim report for the first quarter of 2025, April 2025

“Currently, more than 230 patients are being treated with Emcitate.” (opens the source at this quote in a new tab)

Cited source: None stated in the document.

Egetis Therapeutics (2025) interim report for the first quarter of 2025, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Prevalence (males and the total population): Rare Thyroid Therapeutics International benefit assessment dossier for Germany, April 2025

“eine Prävalenz von 1:70.000 unter Jungen/Männern und folglich eine Prävalenz von 1:140.000 an der Gesamtbevölkerung” (opens the source at this quote in a new tab)

“Vor dem Hintergrund der sehr wenigen beschriebenen Fälle insgesamt und der Meinung klinischer Experten erscheint diese Prävalenz allerdings deutlich zu hoch.” (opens the source at this quote in a new tab)

Cited source: 2.5 Groeneweg 2020

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 25 of 67 (opens in a new tab)

Sources the company cites

European medicines agency 2018

Reference as the company lists it

“European Medicines Agency (EMA). Public summary of opinion on orphan designation Tiratricol for the treatment of Allan-Herndon-Dudley syndrome. Stand: 08.01.2018. 2018.” (opens the source at this quote in a new tab)

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 12 of 67 (opens in a new tab)

Passage carrying the figure

“At the time of designation, Allan-Herndon-Dudley syndrome affected less than 0.01 in 10,000 people in the European Union (EU).” (opens the source at this quote in a new tab)

European Medicines Agency (2018) public summary of opinion on orphan designation EU/3/17/1945, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 1 of 4 (opens in a new tab)

Kubota 2022

Reference as the company lists it, Rare Thyroid Therapeutics International benefit assessment dossier Module 3 A

“Kubota M, Yakuwa A, Terashima H, Hoshino H. A nationwide survey of monocarboxylate transporter 8 deficiency in Japan: Its incidence, clinical course, MRI and laboratory findings. Brain Dev. 2022;44(10):699-705.” (opens the source at this quote in a new tab)

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 33 of 67 (opens in a new tab)

Reference as the company lists it, Egetis Therapeutics press release, November 10, 2023

“Kubota, M. et al. (2022) A nationwide survey of monocarboxylate transporter 8 deficiency in Japan: Its incidence, clinical course, MRI and laboratory findings. Brain Development 44:699-705.” (opens the source at this quote in a new tab)

Egetis Therapeutics (2023) press release, November 10, 2023, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Passage carrying the figure, prevalence in Japan

“We estimated the prevalence of MCT8 deficiency to be 1 in 1,890,000” (opens the source at this quote in a new tab)

Kubota 2022, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Passage carrying the nearest figure, number of patients in Japan

“we sent case surveys to 31 hospitals (45 cases of genetic diagnosis)” (opens the source at this quote in a new tab)

Kubota 2022, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Egetis therapeutics 2025

Reference as the company lists it

“Egetis Therapeutics AB. Daten und Berechnung der Anzahl von GKV-Patienten in der Zielpopulation. 2025.” (opens the source at this quote in a new tab)

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 34 of 67 (opens in a new tab)

Passage carrying the figure

Not quoted: Unpublished company data and calculation; no public document exists.

Statistisches bundesamt 2024

Reference as the company lists it

“Statistisches Bundesamt (Destatis). Bevölkerungsstand 2011 bis 2023 in Deutschland [Stand: 14. Juni 2024]. 2024.” (opens the source at this quote in a new tab)

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 34 of 67 (opens in a new tab)

Passage carrying the figure

Not quoted: A population statistics table that a person must query; the dossier names no address, and the table gives the population of Germany to which the company applies its prevalence range, with no prevalence figure of its own.

Groeneweg 2020

Reference as the company lists it

“Groeneweg S, van Geest FS, Abaci A, Alcantud A, Ambegaonkar GP, Armour CM, et al. Disease characteristics of MCT8 deficiency: an international, retrospective, multicentre cohort study. Lancet Diabetes Endocrinol. 2020;8(7):594-605.” (opens the source at this quote in a new tab)

Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A, quoted passage highlighted (opens the full-size image in a new tab)
Captured from page 31 of 67 (opens in a new tab)

Passage carrying the figure

Not quoted: The main text of the article on PMC states no prevalence figure; the calculation of 1:70,000 males that the dossier attributes to the authors was not found there, and the supplementary appendix was not read.

Iwayama 2021

Reference as the company lists it

“Iwayama, H. et al (2021) Regional Difference in Myelination in Monocarboxylate Transporter 8 Deficiency: Case Reports and Literature Review of Cases in Japan. Front. Neurol. 12:657820.” (opens the source at this quote in a new tab)

Egetis Therapeutics (2023) press release, November 10, 2023, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

Passage carrying the figure

“We identified 36 patients with MCT8 deficiency from 25 families reported from Japan.” (opens the source at this quote in a new tab)

Iwayama 2021, quoted passage highlighted (opens the full-size image in a new tab)
Captured from the passage on the page (opens in a new tab)

References

  1. Egetis Therapeutics (2023) press release, December 13, 2023 (opens in a new tab)
  2. Egetis Therapeutics (2023) press release, November 10, 2023 (opens in a new tab)
  3. Egetis Therapeutics (2025) interim report for the first quarter of 2025 (opens in a new tab)
  4. Egetis Therapeutics (2026) annual report for 2025 (opens in a new tab)
  5. Egetis Therapeutics (2026) interim report for the first quarter of 2026 (opens in a new tab)
  6. Egetis Therapeutics (2026) interim report for the second quarter of 2026 (opens in a new tab)
  7. Egetis Therapeutics (2026) press release, January 29, 2026 (opens in a new tab)
  8. Egetis Therapeutics (2026) second quarter 2026 webcast presentation (opens in a new tab)
  9. Egetis Therapeutics (2026) website, MCT8 deficiency page for healthcare professionals (opens in a new tab)
  10. European Medicines Agency (2018) public summary of opinion on orphan designation EU/3/17/1945 (opens in a new tab)
  11. Groeneweg 2020 (opens in a new tab)
  12. Iwayama 2021 (opens in a new tab)
  13. Kubota 2022 (opens in a new tab)
  14. Rare Thyroid Therapeutics International (2025) benefit assessment dossier Module 3 A (opens in a new tab)