Dingxin Gene's Dual-Vector AAV Gene Therapy RRG-OTOF Receives FDA Rare Pediatric Disease and Orphan Drug Designations

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On November 18, 2023 (Eastern Time), RRGENER (Shanghai Dingxin Gene Technology Co., Ltd.) and the research team led by Shu Yilai and Li Huawei from the Eye, Ear, Nose, and Throat Hospital of Fudan University received the Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for their co-developed RRG-OTOF dual-vector AAV to treat OTOF-mediated hearing loss.

On December 21, 2023 (Eastern Standard Time), RRG-OTOF received orphan drug designation (ODD) from the U.S. FDA. This is also the first in vivo gene therapy product independently developed in China for OTOF-mediated hearing loss to receive orphan drug designation from the U.S. FDA.

[About OTOF-Mediated Hearing Loss]

OTOF-Mediated Hearing Loss, Also Known as DNFB9 Congenital Deafness

[About RRG-OTOF]

RRG-OTOF is a gene therapy based on a dual-vector adeno-associated virus (AAV) system, which treats OTOF-mediated hearing loss by delivering the gene encoding otoferlin into the inner hair cells of the cochlea. A single administration of RRG-OTOF in the cochlea allows the AAV dual vectors to enter the same cell, enabling long-term and sustained expression of full-length functional otoferlin protein to restore auditory function.

[About ODD and RPDD]

To incentivize the development of new therapies for rare diseases/pediatric rare diseases, the FDA Office of Orphan Products Development (OOPD) grants Orphan Drug Designation (ODD) to eligible drugs (including biologics) intended for the prevention, treatment, and diagnosis of rare diseases. The FDA Office of Pediatric Therapeutics (OPT) and OOPD jointly confer Rare Pediatric Disease Designation (RPDD) to drugs that meet specific criteria. Drugs granted ODD are entitled to corresponding policy benefits, including a 7-year marketing exclusivity upon approval of the new drug, waiver of NDA/BLA application fees, tax credits for clinical research expenses, and potential exemption from certain clinical data submission requirements, among other incentives. For products receiving RPDD, sponsors can be awarded a Priority Review Voucher (PRV) by the FDA after obtaining approval for their New Drug Application (NDA) or Biologics License Application (BLA). This PRV, once redeemed, can expedite the review process for subsequent NDAs or BLAs of the sponsor’s other products or can be sold to other sponsors, granting them the same priority review rights.