THX Pharma Exploits European Patent License for Batten-1 Until 2041
THX Pharma (Theranexus) and the Beyond Batten Disease Foundation have announced the issuance by the European Patent Office of a patent protecting the therapeutic use of Batten-1 in juvenile Batten disease (CLN3). The company views this issuance as part of a protection strategy based on three distinct pillars.
A Use Patent Exploited Under Worldwide Exclusive License
European patent No. EP 4 188 371 protects the use of miglustat, the active ingredient of Batten-1, in the treatment of the juvenile form of Batten disease (CLN3). It is jointly held by the Beyond Batten Disease Foundation, Baylor College of Medicine (Houston, Texas) and Cardiff University (United Kingdom). Pursuant to the agreement signed in late 2019, the BBDF, which holds the exclusive exploitation rights, has granted THX Pharma an exclusive and worldwide license for the development and commercial exploitation of Batten-1. The patent designates 38 contracting states of the European Patent Convention, including all European Union member states, the United Kingdom, Switzerland and Norway, and confers protection until July 30, 2041.
Protection Based on Three Complementary Pillars
The company describes a protection strategy based on three independent pillars. The therapeutic use patent now issued protects the use of miglustat regardless of pharmaceutical form. It is added to THX Pharma's proprietary formulation patent portfolio, covering a high-concentration oral solution of miglustat intended for pediatric administration. The third pillar is based on orphan drug designations obtained in the United States and the European Union. The FDA's official database lists the designation of miglustat alone in juvenile Batten disease since January 2021, while the European designation was granted by the European Commission in October 2020, on the advice of the European Medicines Agency (EMA). These designations may confer, following the grant of a marketing authorization and subject to applicable regulatory conditions, commercial exclusivity in their respective jurisdictions. The patent issuance comes as applications for authorization of the phase 3 clinical trial Batten-1-02 have just been submitted to the FDA, the MHRA and for the European Union, and is part of the worldwide licensing agreement concluded with Biocodex in February 2026.
