MH-101
Urea cycle disorders
A specialised medicine being advanced for adjunctive management in urea cycle disorders, with a US-funded clinical programme and orphan designation in the United States.
- Indication
- Adjunctive management of hyperammonaemia
- Patient population
- Paediatric and adolescent patients
- Regulatory status
- IND active; UK CTA in preparation
- Geography
- United Kingdom · European Union · United States
- Partner status
- Wholly owned
- Commercial note
- Retained UK and EU rights; US development funded.
Milestones
- US orphan designation granted2024
- First-in-patient dosing initiated2025
Under clinical evaluation. Not approved for marketing in any territory.
