Home/Pipeline/Emcitate® (tiratricol)

Emcitate® (tiratricol)

Monocarboxylate Transporter 8 (MCT8) Deficiency / Allan-Herndon-Dudley Syndrome

Approved / Phase 3Active

Key Facts

Indication
Monocarboxylate Transporter 8 (MCT8) Deficiency / Allan-Herndon-Dudley Syndrome
Phase
Approved / Phase 3
Status
Active
Company

About Egetis Therapeutics

Egetis Therapeutics is an integrated pharmaceutical company executing a de-risked, late-stage orphan drug strategy. Its core achievement is the EU marketing authorization and launch in Germany for Emcitate® (tiratricol), the first-ever treatment for the ultra-rare Allan-Herndon-Dudley Syndrome (MCT8 deficiency), with a US NDA under Priority Review. The company's strategy centers on advancing this asset through global approvals and commercialization while preparing its second program, Aladote®, for pivotal studies, aiming to build a sustainable portfolio of niche therapies.

View full company profile

About Egetis Therapeutics

Egetis Therapeutics is an integrated pharmaceutical company executing a de-risked, late-stage orphan drug strategy. Its core achievement is the EU marketing authorization and launch in Germany for Emcitate® (tiratricol), the first-ever treatment for the ultra-rare Allan-Herndon-Dudley Syndrome (MCT8 deficiency), with a US NDA under Priority Review. The company's strategy centers on advancing this asset through global approvals and commercialization while preparing its second program, Aladote®, for pivotal studies, aiming to build a sustainable portfolio of niche therapies.

View full company profile