Posted by Michael Wonder on 12 Aug 2020
Theranexus and BBDF obtain orphan drug designation and rare paediatric disease designation from the FDA for BBDF-101 for Batten disease
11 August 2020 - Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF) today announced the decision by the FDA to award orphan drug designation and rare paediatric disease designation to the drug candidate BBDF-101 for Batten disease, a rare, fatal, genetic disorder of the nervous system for which there is no treatment.
In late 2019, Theranexus and 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.
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.
