Drug Update · Otolaryngology

FDA Approves lunsotogene parvec for OTOF Hearing Loss: 80% ≤70 dB PTA

July 22, 2026

Approval date

04/23/2026

Primary endpoint result

16/20 (80%) achieved PTA ≤70 dB HL at Week 24

Key secondary endpoint

14/20 (70%) ABR to click ≤90 dB nHL at Week 24

Study name and design

Study DB-OTO-001, ongoing multi-center single-arm

Treated patients

24 patients

Approved dose

7.2 × 10 12 vector genomes (vg) in 0.24 mL per ear (intracochlear infusion)

Clinical Perspective

Surgeons experienced in intracochlear surgery and clinicians who treat pediatric and adult patients with molecularly confirmed biallelic OTOF variants and preserved outer hair cell function should be aware of this new option for severe-to-profound and profound sensorineural hearing loss. The FDA accelerated approval supports that Otarmeni improves hearing sensitivity, with 16/20 (80%) of evaluable patients achieving a PTA ≤70 dB HL at Week 24 in Study DB-OTO-001. Will the required confirmatory trial verify durability of benefit and demonstrate effects on speech development and quality of life in treated patients?

Editorial Team · Otolaryngology

Otarmeni (lunsotogene parvec-cwha) received accelerated approval from the U.S. Food and Drug Administration on April 23, 2026 for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic OTOF variants, preserved outer hair cell function, and no prior cochlear implant in the same ear. The approval was based on Study DB-OTO-001 in which 16/20 (80%) evaluable patients achieved a hearing sensitivity threshold ≤70 dB HL at Week 24 ^[2]^[3]^[1].

Clinical Context

Lunsotogene parvec (Otarmeni) is an adeno-associated virus vector-based gene therapy developed by Regeneron Pharmaceuticals, Inc. Otarmeni is administered by single-dose intracochlear infusion and is indicated for pediatric and adult patients with molecularly confirmed biallelic OTOF variants and preserved outer hair cell function; patients must not have a prior cochlear implant in the same ear ^[1]^[3]. Intracochlear infusion of Otarmeni results in expression of the OTOF gene primarily in inner hair cells to facilitate production of functional otoferlin protein and restore synaptic transmission to the auditory nerve, as shown in animal studies and described in the product labeling ^[3]^[1]. The application was reviewed under the Commissioner’s National Priority Voucher pilot and granted accelerated approval ^[2].

Key Findings

Lunsotogene parvec (Otarmeni) achieved the primary efficacy endpoint of a hearing sensitivity threshold ≤70 dB HL assessed by average pure tone audiometry (PTA) at Week 24 in 16/20 (80%) patients in Study DB-OTO-001 (single-arm) ^[3].
The key secondary endpoint, presence of auditory brainstem response (ABR) to a click stimulus of ≤90 dB nHL at Week 24, was observed in 14/20 (70%) evaluable patients in Study DB-OTO-001 ^[3].
Among the 20 patients with Week 24 data, 9/20 (45%) achieved an average PTA ≤45 dB HL and 3/20 (15%) achieved ≤25 dB HL at Week 24; durability data from a subset of 12 patients at Week 48 showed that all 9 patients who responded at Week 24 and were evaluated at Week 48 maintained their response, and one additional patient converted to response by Week 48 resulting in 10/12 (83%) responders at Week 48 ^[3].

Safety & Tolerability

The most common adverse reactions (incidence ≥5%) included otitis media, vomiting, nausea, dizziness, procedural pain, gait disturbance, and nystagmus ^[1].
Procedure-related risks and adverse reactions named in the prescribing information include vertigo, tinnitus, cerebral spinal fluid leak, ipsilateral facial paresis, ipsilateral change in taste, meningitis, wound infection, mastoiditis, numbness around the ear, blood or fluid collection at the surgical site, and labyrinthitis ^[1].
Clinical-trial experience (Study DB-OTO-001, N=24 treated) also listed transient balance disorder, abnormal otoacoustic emissions, and wound dehiscence among other clinically significant adverse reactions ^[1]^[3].

Study Design

The efficacy basis was an ongoing, multi-center, single-arm study (Study DB-OTO-001; NCT05788536) that enrolled pediatric patients with molecularly confirmed OTOF gene-associated profound sensorineural hearing loss and evidence of preserved outer hair cell activity; 24 patients received OTARMENI and 20 patients had evaluable Week 24 efficacy data. The primary efficacy endpoint was achievement of a hearing sensitivity threshold ≤70 dB HL by average PTA at Week 24. The FDA granted accelerated approval on April 23, 2026; continued approval may be contingent upon verification of clinical benefit in the confirmatory trial ^[3]^[2]^[1].

FAQ

Q1 What is lunsotogene parvec (Otarmeni) approved for?

Otarmeni is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. This indication was approved under accelerated approval based on improvement in hearing sensitivity assessed by average PTA at Week 24, and continued approval may be contingent on confirmatory trial results.

Q2 How does lunsotogene parvec (Otarmeni) work?

Otarmeni is an adeno-associated virus vector-based gene therapy designed for intracochlear infusion. Intracochlear administration results in expression of the OTOF gene primarily in inner hair cells, facilitating production of functional otoferlin protein and restoring synaptic transmission to the auditory nerve according to nonclinical and clinical data summarized in the prescribing information. Mechanistic and sustained otoferlin expression data supported the approval.

Q3 What is the recommended dose of lunsotogene parvec (Otarmeni)?

The recommended dose of OTARMENI for each ear is 7.2 × 10 12 vector genomes (vg) in a total volume of 0.24 mL, administered by a single-dose intracochlear infusion. OTARMENI should be administered by a surgeon experienced in intracochlear surgery and trained in the administration procedure; administer bilateral OTARMENI in a single surgical session if applicable. For prophylaxis against inflammatory and immunological responses, systemic oral corticosteroids equivalent to prednisone at 1 mg/kg/day the day of infusion, maintained for two weeks and tapered over the following two weeks (not to exceed 60 mg/day), are recommended per the label. Clinicians should consult current prescribing information for complete dosing guidance.

Q4 What are the most common side effects of lunsotogene parvec (Otarmeni)?

Adverse reactions reported in clinical experience (Study DB-OTO-001 and in the prescribing information) include otitis media, vomiting, nausea, dizziness, procedural pain, gait disturbance, and nystagmus among those occurring in ≥5% of patients. Procedure-related risks specifically named include vertigo, tinnitus, cerebral spinal fluid leak, ipsilateral facial paresis, ipsilateral change in taste, meningitis, wound infection, mastoiditis, numbness around the ear, blood or fluid collection at the surgical site, and labyrinthitis. Clinicians should monitor for surgical and procedure-related complications and consult the full prescribing information for comprehensive safety details and management guidance. Clinicians should consult current prescribing information for complete dosing guidance.

FDA Approves lunsotogene parvec for OTOF Hearing Loss: 80% ≤70 dB PTA

FDA Approves lunsotogene parvec for OTOF Hearing Loss: 80% ≤70 dB PTA

Clinical Perspective

Clinical Context

Key Findings

Safety & Tolerability

Study Design

FAQ

References

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