Biologics

Regeneron Gene Therapy Improved Rare Hearing Loss

Regeneron Pharmaceuticals says new clinical data show its investigational gene therapy DB-OTO has restored meaningful hearing in nearly all children treated for genetic hearing loss caused by OTOF gene variants. The company plans to submit a BLA later this year after discussing its options with the agency.

The data were published in the New England Journal of Medicine and presented at the American Academy of Otolaryngology–Head and Neck Surgery Foundation annual meeting. It says that 11 of 12 participants in its pivotal CHORD trial showed clinically significant hearing improvements, with three achieving normal hearing sensitivity. Among the eight participants with longer follow-up, hearing gains remained stable or continued to improve, it adds.

The ongoing Phase 1/2 CHORD trial is evaluating DB-OTO in infants, children, and adolescents (aged 10 months to 16 years) with profound hearing loss caused by OTOF gene mutations, the company says. Participants receive a single intracochlear infusion of the dual AAV vector gene therapy, which delivers a working copy of the OTOF gene to enable production of the otoferlin protein essential for sound transmission in the inner ear.

Regeneron notes that no treatment-related adverse events have been reported. Two serious events — one surgical complication and one related to vaccination — were found unrelated to DB-OTO, it says, adding that temporary vestibular symptoms, such as dizziness and nausea, resolved fully.

Otoferlin-related hearing loss is an ultra-rare congenital condition affecting about 20 to 50 newborns annually in the United States. Children with this mutation are born with profound hearing loss due to the absence of functional otoferlin protein, which disrupts communication between sensory cells and the auditory nerve.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome