We are excited to announce the following major milestone for BBDF!
The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101. The aim of the program is to facilitate the development of new drugs and biological products for the prevention and treatment of rare pediatric diseases. These designations provide accelerated review for approval, support with the FDA regulatory process and at least seven years of post-approval protection, as well as exemption from filing fees that normally have to be paid to the FDA. These designations do not impact the clinical trial, but are beneficial once the therapy receives New Drug Approval. We are grateful to Theranexus for their continued partnership and commitment to initiating a clinical trial for BBDF-101 as quickly as possible.
THERANEXUS AND BBDF OBTAIN ORPHAN DRUG DESIGNATION (ODD) AND RARE PEDIATRIC DISEASE DESIGNATION (RPDD) FROM THE FOOD AND DRUG ADMINISTRATION (FDA) FOR BBDF-101 FOR BATTEN DISEASE
- Orphan Drug Designation (ODD) is a status that provides seven years of additional post-approval protection and exemption from filing fees
- Rare Pediatric Disease Designation (RPDD) qualifies the sponsor at the time of registration for a salable, transferable priority review voucher which can be used to speed up the approval process for another drug candidate
Lyon, August 28, 2020 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF) today announced the decision by the Food and Drug Administration (FDA) to award 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.