Lunsotogene parvec

Lunsotogene parvec
Clinical data
Trade namesOtarmeni
Other namesDB-OTO, lunsotogene parvec-cwha
AHFS/Drugs.comotarmeni
License data
Routes of
administration		Intracochlear infusion
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII

Lunsotogene parvec, sold under the brand name Otarmeni, is a gene therapy treatment designed to reverse nonsyndromic deafness caused by a rare mutation in the OTOF gene. It is an adeno-associated virus vector-based gene therapy.[2] The drug was developed under the investigational name DB-OTO by Decibel Therapeutics, which was bought out by Regeneron in 2023. The drug was approved by the US Food and Drug Administration in 2026. It is the first approved gene therapy to target hearing loss. The drug uses an adeno-associated virus to deliver DNA encoding otoferlin that is regulated by a hair cell–specific promoter.[3][4][5][6][7][8][9][10][11][12][13][14]

Medical uses

Lunsotogene parvec is indicated for the treatment of people 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.[2]

Society and culture

Lunsotogene parvec was approved for medical use in the United States in April 2026.[2]

Names

Lunsotogene parvec is the international nonproprietary name.[15]

Lunsotogene parvec is sold under the brand name Otarmeni.[2]

References

  1. ^ "Otarmeni- lunsotogene parvec injection, solution". DailyMed. 27 April 2026. Retrieved 10 May 2026.
  2. ^ a b c d "Otarmeni". U.S. Food and Drug Administration. 23 April 2026. Retrieved 8 May 2026. Public Domain This article incorporates text from this source, which is in the public domain.
  3. ^ Stein R (23 April 2026). "The FDA gives the green light to the first gene therapy for deafness". NPR. Retrieved 24 April 2026.
  4. ^ Stein R (22 April 2026). "Gene therapy for a rare type of deafness shows lasting results". NPR. Retrieved 24 April 2026.
  5. ^ Lovelace B (23 April 2026). "FDA approves first gene therapy for genetic hearing loss". NBC. Retrieved 24 April 2026.
  6. ^ Kolata G (23 April 2026). "New Gene Therapy Enables Children With a Rare Form of Deafness to Hear". The New York Times. Retrieved 24 April 2026.
  7. ^ Roy S (23 April 2026). "Regeneron wins FDA approval for first gene therapy for genetic hearing loss". Reuters. Retrieved 24 April 2026.
  8. ^ Valayannopoulos V, Bance M, Carvalho DS, Greinwald JH, Harvey SA, Ishiyama A, et al. (March 2026). "DB-OTO Gene Therapy for Inherited Deafness". New England Journal of Medicine. 394 (11): 1074–1083. doi:10.1056/NEJMoa2400521. PMID 41085057.
  9. ^ Le Prell CG (August 2023). "Preclinical prospects of investigational agents for hearing loss treatment". Expert Opinion on Investigational Drugs. 32 (8): 685–692. doi:10.1080/13543784.2023.2253141. PMID 37695693. S2CID 261694986.
  10. ^ Philippidis A (December 2020). "Bayer, Novartis, Pfizer Raise Big Pharma's Stake in Gene Therapy". Human Gene Therapy. 31 (23–24): 1221–1223. doi:10.1089/hum.2020.29142.bfs. PMID 33337268. S2CID 229316419.
  11. ^ Philippidis A (1 January 2023). "StockWatch: Regeneron's All Ears for Hearing Loss Drug Developer: With planned up-to-$213M buyout, Decibel joins a growing number of biotechs focused on treating ear disorders to find a buyer". GEN Edge. 5 (1): 542–548. doi:10.1089/genedge.5.1.105. S2CID 265960410.
  12. ^ Jiang L, Wang D, He Y, Shu Y (April 2023). "Advances in gene therapy hold promise for treating hereditary hearing loss". Molecular Therapy. 31 (4): 934–950. doi:10.1016/j.ymthe.2023.02.001. PMC 10124073. PMID 36755494.
  13. ^ Hayward E (9 May 2024). "Toddler born deaf can hear after world-first gene therapy". The Times. Retrieved 9 May 2024.
  14. ^ Incorvaia D (23 April 2026). "Regeneron ushers in new genetic medicine era with groundbreaking gene therapy approval". Fierce Pharma. Retrieved 24 April 2026.
  15. ^ World Health Organization (2025). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 94". WHO Drug Information. 39 (3). hdl:10665/383022.
  • Clinical trial number NCT05788536 for "A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/​Infants With Hearing Loss Due to Otoferlin Mutations (CHORD)" at ClinicalTrials.gov


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