FDA Grants Rare Pediatric Disease Designation to Theranexus and BBDF for Batten Disease Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Theranexus and Beyond Batten Disease Foundation Rare Pediatric Disease designation to their experimental therapy BBDF-101 for Batten disease, a rare and fatal neurological disorder.

The agency also granted the therapy orphan drug designation.

Juvenile Batten disease is part of a group of disorders referred to as neuronal ceroid lipofuscinoses. The condition, also known as CLN3 disease, is a genetic disorder that primarily affects the nervous system. Children with the condition develop normally until ages 5 to 6, when progressive vision loss becomes noticeable. Personality changes and behavioral issues follow. Children eventually go blind, suffer seizures, and they experience a decline in speech and progressive loss of motor coordination. Eventually they become wheelchair-bound, bed-ridden, and die in their late teens to late twenties.

Beyond Batten Disease Foundation (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. In late 2019, Theranexus and BBDF signed an agreement granting Theranexus an exclusive, global license agreement for the development and commercial use of BBDF-101 for the treatment of juvenile Batten disease.

“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 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.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes BBDF-101 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Photo: Franck Mouthon, chairman and CEO of Theranexus

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.