“We are very pleased by the FDA’s recent approval of the preclinical development plan for BBDF-101, as this paves the way for the initiation of clinical trials which we all hope will deliver a therapeutic response for children and young adults with this terrible disease” said Craig Benson, chair of the BBDF board of directors, in a press release.
BBDF-101 is designed to help cells clear out the toxic waste that accumulates over the course of Batten disease, due to inefficient lysosomes, a cellular recycling and waste disposal organelle. It does this by activating the transcription factor EB (TFEB) gene, which controls lysosome production. This is expected to slow the disorder’s progression.
“We are delighted about this latest positive meeting with the FDA. The result is the continuation of an optimized preclinical development plan to initiate clinical trials in patients with Batten disease starting in 2021” said Franck Mouthon, chairman and CEO of Theranexus.
Theranexus and the BBDF partnered to develop the investigational new therapy in late 2019. Their proposed trial will evaluate the compound’s safety, and compare its effectiveness to the natural progression of Batten disease among adolescent/adult patients and pediatric participants.
The trial will begin with the enrollment of six adolescent/adult patients, who will be administered BBDF-101 in escalating doses, with tolerability and pharmacokinetics (how a compound moves through the body) established over five months. Participants will undergo follow-ups for safety. After tolerability and pharmacokinetics measurements, 30 pediatric patients will be enrolled and assessed for two years for disease progression.
Orphan drug designation grants a medication’s developer extra market exclusivity upon approval and exemption from paying certain regulatory fees.
Rare pediatric disease designation awards a developer with a voucher that can be used to obtain an accelerated review of its product, taking place within six months, down from the usual one year. This voucher can be exchanged alternatively for an accelerated review of a different treatment.
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