LYON, France—Theranexus and the Beyond Batten Disease Foundation (BBDF) have announced that the U.S. Food and Drug Administration (FDA) has awarded both Orphan Drug and Rare Pediatric Disease designations to the drug candidate BBDF-101. BBDF-101 is intended to treat Batten disease, a fatal genetic disorder of the nervous system for which there is no current treatment.
“We are delighted to have obtained Orphan Disease Designation (ODD) and Rare Pediatric Disease Designation (RPDD). This marks a new milestone for Theranexus and BBDF in the development of the drug candidate BBDF-101,” said Franck Mouthon, chairman and CEO of Theranexus. “These new designations will speed up the approval process and provide at least seven years of post-approval protection and exemption from filing fees, as well as qualifying Theranexus upon approval of BBDF-101 for an assignable and transferable priority review voucher upon the registration of BBDF-101 that can be used to speed up the approval process for any other drug.”
Theranexus and BBDF signed an agreement in late 2019, which granted Theranexus an exclusive, global license agreement for the development and commercial use of BBDF-101 for the treatment of juvenile Batten disease. Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses. The BBDF has funded research aimed at identifying and validating BBDF-101, which is a proprietary combination of drugs based on the synergistic effect of two active ingredients.
“We are delighted to have been awarded Orphan Drug Designation for BBDF-101 by the FDA. This is a sign of recognition for Batten disease and raises hopes for children and teens with this orphan disorder. I would like to say a big thank you to the entire BBDF team involved in the FDA submission, as well as to our donors, volunteers and the partner families of the foundation, without whom none of this would have been possible,” added Craig Benson, chair of the board of directors for BBDF.
After discussions with the FDA, Theranexus is preparing to launch a preclinical trial in order to confirm the preclinical safety of BBDF-101 over a long exposure time, with the aim of supplementing the available data. The company plans to launch a clinical program in 2021.