生产企业：THX Pharma (Theranexus), Exeltis (Insud Pharma group)
TX01 is an investigational paediatric formulation developed by THX Pharma (Theranexus) and Exeltis for the treatment of Niemann-Pick type C disease and Gaucher disease. The EMA's Paediatric Committee (PDCO) issued a positive Opinion on the Paediatric Investigation Plan (PIP) in June 2026, enabling a Paediatric Use Marketing Authorisation (PUMA) submission planned for Q1 2027, with commercial launch in Europe targeted for 2027.
TX01 is an investigational paediatric formulation developed by THX Pharma (Theranexus) and Exeltis for the treatment of Niemann-Pick type C disease and Gaucher disease. The EMA's Paediatric Committee (PDCO) issued a positive Opinion on the Paediatric Investigation Plan (PIP) in June 2026, enabling a Paediatric Use Marketing Authorisation (PUMA) submission planned for Q1 2027, with commercial launch in Europe targeted for 2027.
TX01 is being developed for the treatment of Niemann-Pick type C disease and Gaucher disease, two rare, progressive, and severely debilitating lysosomal storage disorders for which paediatric therapeutic options remain limited. The EMA Paediatric Committee (PDCO) issued a positive Opinion on the Paediatric Investigation Plan (PIP) for TX01 in Niemann-Pick type C disease in June 2026, with a Paediatric Use Marketing Authorisation (PUMA) submission planned for Q1 2027.
共 2 项相关疾病
