THX Pharma and Exeltis receive a positive opinion from the EMA for TX01

The Pediatric Investigation Plan (PIP) for TX01 for Niemann-Pick disease type C has been approved by the EMA's Pediatric Committee. This step paves the way for a Pediatric Marketing Authorisation (PUMA) application, guaranteeing up to 10 years of commercial protection in Europe.

Expected in early 2027, the PUMA application represents a major step forward in the partnership between THX Pharma and Exeltis. With this positive opinion, the pediatric development of TX01 progresses, confirming compliance with European regulatory requirements.

The PUMA procedure strengthens the commercial strategy, offering protection similar to that of orphan drugs and simplifying market access. TX01 thus aims to address the lack of treatments for rare lysosomal diseases.