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.
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 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.
“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,” explains Craig Benson, Chair of the BBDF Board of Directors.
“We are delighted to have obtained Orphan Disease Designation and Rare Pediatric Disease Designation (RPDD). This marks a new milestone for Theranexus and BBDF in the development of the drug candidate BBDF-101. 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 on registration of BBDF-101 for a salable and transferable priority review voucher that can be used to speed up the approval process for any other drug,” continues Franck Mouthon, Theranexus Chairman and CEO.
The legal status of orphan designation in the United States was provided by the Orphan Drug Act of 4 July 1983. It qualifies the Company for accelerated review of its drug candidate 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.
The FDA defines rare pediatric diseases as diseases that are serious or life threatening and primarily affect individuals aged under 18, with fewer than 200,000 cases in the United States. 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. When the FDA awards Rare Pediatric Disease Designation, the sponsor of the trial qualifies for a priority review voucher, which may be sold to another company or transferred to another drug candidate. In such case the length of the approval procedure is also considerably shortened, generally taking just 6 months rather than 12 months on average, which gives a very high value to this priority review voucher.
After discussions with the FDA, Theranexus is currently preparing to launch a BBDF-101 preclinical trial to confirm the preclinical safety of BBDF-101 over a long exposure time, with the aim of supplementing the data already available so that the clinical program can be launched in 2021.
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BEYOND BATTEN DISEASE FOUNDATION
Mary Beth Kiser
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