Hearing gains are not the endpoint: What should clinicians watch after OTOF gene therapy?

By Alpana Mohta, MD, DNB, FEADV, FIADVL, IFAADFact-checked by Barbara BekieszPublished June 18, 2026

Industry Buzz

Follow-up should be long-term and multidisciplinary. These children need audiology, otology, speech-language therapy, genetics, developmental pediatrics, and school-support monitoring, not just repeat ABRs.

—Jeremy Woods, MD

A first-line gene therapy for hearing loss caused by mutations in the OTOF gene has arrived. The therapy, lunsotogene parvec-cwha (Otarmeni; approved April 2026) was approved based on marked improvements in hearing sensitivity—but hearing thresholds are only one piece of the story.

What clinicians want to know now is whether those gains help a child communicate more effectively, develop language, participate in school, and navigate everyday life.^[] Ongoing follow-up will ideally show that those benefits are durable and translate into meaningful outcomes such as speech development, language acquisition, and quality of life.

Looking beyond hearing thresholds

A child may demonstrate better hearing on testing yet continue to face challenges with speech understanding, language development, listening in noisy settings, classroom engagement, or social interactions. These functional outcomes are often what matter most to families.

In younger children, especially infants and toddlers, progress may be better reflected through developmental milestones, caregiver reports, and speech-language evaluations than through standard audiometric testing alone.

Tracking real-world progress

"After OTOF gene therapy, the outcomes that matter most are functional: speech perception, spoken-language growth, hearing in noise, school readiness, quality of life, caregiver-reported communication, and whether the child still needs hearing devices," says Jeremy Woods, MD, board-certified in pediatrics and medical genetics and genomics, and director of Valley Children’s Healthcare’s Prenatal Diagnostic Center and a physician in the hospital’s Medical Genetics and Metabolism Clinic.

Safety also remains part of follow-up. Dr. Woods says, “Follow-up should be long-term and multidisciplinary. These children need audiology, otology, speech-language therapy, genetics, developmental pediatrics, and school-support monitoring, not just repeat ABRs."

Clinicians must also watch for surgical and treatment-related complications, including vertigo, tinnitus, cerebrospinal fluid leak, facial weakness, meningitis, wound infection, mastoiditis, and labyrinthitis. ^[]Adverse events seen in clinical studies included otitis media, nausea, vomiting, dizziness, procedural pain, gait disturbance, and nystagmus.^[]

Dr. Woods offered some counseling points for families. “The most important counseling point is that timing matters, but progress is not instantaneous. Families should expect close monitoring over months, uncertainty about durability and degree of benefit, and a continued need to protect language development while hearing outcomes evolve," he says.

For clinicians, the promise of a new gene therapy for genetic pediatric hearing loss is best understood as the beginning of a longer developmental trajectory. Improved hearing sensitivity may create a critical opening for children with OTOF-related hearing loss, but the full value of treatment will depend on how well those auditory gains support communication, language growth, learning, and family life over time.

That makes early identification, timely referral, and coordinated follow-up especially important. As more children are treated and followed longitudinally, clinicians will be better able to define which patients benefit most, how durable the response is, and how gene therapy can be integrated with audiology, speech-language support, educational planning, and family-centered care. For now, Otarmeni represents a meaningful step forward—one that gives clinicians and families a new reason to act early while continuing to measure success in the outcomes that matter most.

SHARE THIS ARTICLE