FDA Names Investigational Therapy BBDF-101 Orphan Drug, Rare Pediatric Disease

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation and rare pediatric disease designation to BBDF-101, an investigational treatment for Batten disease.

Theranexus is developing BBDF-101 — which seeks to slow the progression of the inherited neurological condition — pursuant to an agreement with the Beyond Batten Disease Foundation (BBDF).

“We are delighted to have obtained Orphan Disease Designation (ODD) and Rare Pediatric Disease Designation (RPDD),” Franck Mouthon, chairman and CEO at  Theranexus said in a press release.

“This marks a new milestone for Theranexus and BBDF in the development of the drug candidate BBDF-101,” Mouthon said.

Craig Benson, chair of the BBDF board of directors, concurred, adding: “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,” he added.

BBDF-101 is a proprietary combination therapy that is intended to help cells clear toxic waste material by activating a molecule known as transcription factor EB (TFEB), thereby slowing the progression of juvenile Batten disease (CLN3).

Theranexus is preparing to launch a BBDF-101 preclinical trial to assess the therapy’s safety over a long exposure time, with plans to begin clinical development next year.

The clinical study will compare BBDF-101’s effectiveness in adolescent/adult and pediatric groups, to the natural progression of Batten as documented in previous groups of patients. Texas Children’s Hospital will be the lead investigation center for the trial, which also will assess the therapy’s safety and pharmacokinetics — how the medicine moves through the body.

Orphan drug designation is given to therapies with the potential to treat rare diseases; rare pediatric disease designation is granted to potential treatments for rare diseases that mainly affect children. For both designations, a rare disease is defined as one that affects fewer than 200,000 people in the U.S.

Winning orphan drug designation gives a therapy’s developer — in this case, Theranexus — extra market exclusivity if the treatment is approved. It also exempts the developer from paying certain filing fees.

Rare pediatric disease designation qualifies the therapy for an accelerated review — typically six months, rather than a year — and the developer is given a voucher that can be exchanged for an accelerated review of a different treatment.

“These new designations will speed up the approval process,” Mouthon said.