Orphan and Rare Pediatric Disease status for Theranexus' BBDF-101

12 August 2020
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The US Food and Drug Administration has awarded Orphan Drug designation (ODD) and Rare Pediatric Disease designation (RPDD) to the drug candidate BBDF-101 for Batten disease, a rare, fatal, genetic disorder of the nervous system for which there is no treatment, under development by French CNS specialist Theranexus (Euronext: ALTHX).

Announced late Tuesday, the news pushed Theranexus’ shares up 6.4% to 10.80 euros in morning trading today.

In late 2019, Theranexus and Texas, USA-based non-profit organization Beyond Batten Disease Foundation (BBDF) signed an agreement granting Theranexus an exclusive, global license agreement for the development and commercial use of drug candidate BBDF-101 for the treatment of juvenile Batten disease. Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs). BBDF funded research aimed at identifying and validating BBDF-101, a proprietary combination of drugs based on the synergistic effect of two active ingredients, like the other Theranexus drug candidates already in clinical development.

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