By VETTAPHARMA reporter – Derek Roche: Regeneron Pharmaceuticals reported that the European Medicines Agency accepted the Marketing Authorisation Application for otarmenitmgene lunsotogene parvovec, an investigational gene therapy for children with hearing loss caused by OTOF gene mutations.
The therapy is being developed for treatment of autosomal recessive deafness 9 (DFNB9), a rare inherited form of profound hearing loss resulting from mutations in the OTOF gene, which affects transmission of sound signals in the inner ear.
The filing acceptance was supported by clinical data evaluating hearing restoration and auditory response following single-dose administration of the gene therapy directly into the cochlea. The programme demonstrated improvements in hearing response and speech perception in treated pediatric patients.
Otarmenitmgene lunsotogene parvovec uses an adeno-associated virus (AAV) vector designed to deliver a functional copy of the OTOF gene to cochlear cells, aiming to restore otoferlin protein expression and hearing function.
The regulatory milestone reflects Regeneron’s strategy of expanding into genetic medicines and rare disease therapies, particularly in areas with significant unmet medical need and limited treatment options.
For children with DFNB9 hearing loss, the investigational therapy may provide a potential disease-modifying treatment approach aimed at restoring hearing function rather than relying solely on supportive interventions such as cochlear implants.
From a commercial perspective, successful approval could position the therapy among the first gene therapies targeting inherited hearing loss, supporting future growth opportunities in rare genetic disorders and precision medicine.
Quick FAQs
1. What is otarmenitmgene lunsotogene parvovec being developed for?
The investigational gene therapy is being developed for children with hearing loss caused by OTOF gene mutations associated with DFNB9.
2. What causes DFNB9 hearing loss?
DFNB9 is caused by mutations in the OTOF gene, which disrupt sound signal transmission in the inner ear.
3. How does the therapy work?
The therapy uses an AAV vector to deliver a functional copy of the OTOF gene directly into cochlear cells.
4. Why is the EMA filing important?
EMA acceptance advances the therapy toward potential approval as a treatment for inherited hearing loss in children.
