Drugs / DB-OTO
DB-OTO
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | prelingual non-syndromic genetic hearing loss | — | “the Company submitted a BLA to the FDA for DB-OTO (an AAV-based gene therapy) for the treatment of profound genetic hearing loss” regeneron.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05788536 | Jun 2023 → Feb 2029 expected | nonsyndromic genetic hearing loss | Regeneron Pharmaceuticals | Recruiting | Met primary |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2026-05-22 | Regeneron Pharmaceuticals | Regulatory Otarmeni™ (lunsotogene parvec) Receives EMA Filing Acceptance for Genetic Hearing Loss regeneron.com ↗ |
| 2026-04-23 | Regeneron Pharmaceuticals | Regulatory Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S. regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni™ (lunsotogene parvec-cwha), the first gene therapy and second new molecular entity approved under the FDA Commissioner’s National Priority Voucher program. |
| 2025-10-12 | Regeneron Pharmaceuticals | Results DB-OTO Results in the New England Journal of Medicine Showcase Dramatic and Sustained Improvements in Hearing and Speech Perception in Children with Profound Genetic Hearing Loss regeneron.com ↗
These latest results from the pivotal CHORD trial show 11 out of 12 participants have experienced clinically meaningful hearing improvements, including 3 who achieved normal hearing levels. |
| 2025-02-24 | Regeneron Pharmaceuticals | Results Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children with Profound Genetic Hearing Loss in CHORD Trial regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced updated data for the investigational gene therapy DB-OTO from the Phase 1/2 CHORD trial in 12 children who have profound genetic hearing loss due to variants of the otoferlin (OTOF) gene. |
| 2024-05-08 | Regeneron Pharmaceuticals | Results Latest DB-OTO Results Show Dramatically Improved Hearing to Normal Levels in a Child with Profound Genetic Deafness within 24 Weeks and Initial Hearing Improvements in a Second Child at 6 Weeks regeneron.com ↗ |
| 2024-04-22 | Regeneron Pharmaceuticals | Results Regeneron to Highlight Advances in Genetic Medicine Research at American Society of Gene and Cell Therapy (ASGCT) regeneron.com ↗
The company will also present updated data from the Phase 1/2 CHORD trial investigating DB-OTO in children with profound genetic hearing loss due to mutations of the otoferlin gene. |
| 2023-10-26 | Regeneron Pharmaceuticals | Results Regeneron Shares Preliminary Results Showing Gene Therapy Improves Auditory Responses in Child with Profound Genetic Hearing Loss regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced preliminary, positive safety and efficacy results from the first patient (<2 years of age) dosed in the Phase 1/2 CHORD trial investigating otoferlin gene therapy (DB-OTO) in children with profound genetic hearing loss due to mutations of the otoferlin gene. |
All press releases naming this drug 7 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | DB-OTO | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05788536 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “DB-OTO is a dual adeno-associated virus 1 gene therapy” PMID 41085057 ↗ Oct 2025 |
| Route | Other | “received an intracochlear infusion of DB-OTO” PMID 41085057 ↗ Oct 2025 |
| Target | OTOF | “delivers human OTOF complementary DNA” PMID 41085057 ↗ Oct 2025 |