Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced updated data for its investigational gene therapy DB‑OTO, treating otoferlin‑related profound hearing loss, which was published in the New England Journal of Medicine and presented at the American Academy of Otolaryngology‑Head and Neck Surgery (AAO‑HNSF) meeting.
The data demonstrate that 11 of 12 participants experienced clinically meaningful hearing improvements, with 3 achieving normal hearing levels; 8 participants with longer follow‑up maintained or improved hearing, and all 3 participants who completed speech assessments showed significant gains, including one who could identify one‑ and two‑syllable words without visual cues and respond to distant sounds in noisy environments.
DB‑OTO has already received Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations from the U.S. Food and Drug Administration, and the company plans to file a U.S. regulatory application later this year, positioning the therapy as a potential first‑in‑class product in a rare disease market.
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