OTOF Hearing Loss Gene Therapy Granted Orphan Drug Designation

Sensorion’s OTOF-GT, an investigational gene therapy intended for the treatment of otoferlin (OTOF) gene-mediated hearing loss, has been granted orphan drug designation (ODD) by the FDA.¹

OTOF-GT functions by delivering OTOF via a dual adeno-associated virus (AAV) vector for assembly in place.² It previously received rare pediatric disease designation in early November. Sensorion has indicated that it is still on track to submit a clinical trial application for the therapy in the first half of next year.¹

“We are really pleased to have received this significant regulatory feedback from the FDA, following the agency’s recent award of rare pediatric disease designation for OTOF-GT,” Géraldine Honnet, chief medical officer, Sensorion, said in a statement regarding the news.¹ “Orphan drug designation will support us in advancing our development program, a gene therapy which offers the potential to help patients with a condition for which there are currently no approved curative therapies. We are excited to have achieved this milestone and remain highly focused on the development of our most promising candidates to produce life-changing therapies to restore, treat and prevent hearing loss disorders.”

OTOF-GT is one of several gene therapies currently in development for OTOF-mediated hearing loss. Decibel Therapeutics’ and Regeneron Pharmaceuticals’ DB-OTO, another investigational AAV dual-vector therapy, received clearance from the FDA for its investigational new drug (IND) application in October of this year.^3,4 The phase 1/2 clinical trial, which will evaluate DB-OTO in pediatric patients, is expected to begin in the first half of next year.⁵ Decibel Therapeutics also announced submissions of clinical trial applications in the United Kingdom and Spain in November.

In September of this year, Akouos's AK-OTOF, which is also an investigational dual AAV vector-based gene therapy intended for the treatment of OTOF-mediated hearing loss, received clearance of its own IND application from the FDA.⁶ The company expects the phase 1/2 clinical trial enabled by the clearance to be global, but initially activated in the United States, with activations in other countries to follow later.⁷ Akouosis in the process of being acquired by Eli Lilly and Company, with the acquisition expected to be consummated by December 1, 2022.⁸

In 2021, DB-OTO and AK-OTOF were both granted ODD and rare pediatric disease designations by the FDA.^3,4,6 In addition to its FDA ODD and rare pediatric disease designation, OTOF-GT was previously granted ODD by the European Commission in October of this year.²

REFERENCES

1. Sensorion announces US FDA grants rare pediatric disease designation to OTOF-GT for the treatment of otoferlin gene-mediated hearing loss. News release. Sensorion. November 30, 2022.https://s27.q4cdn.com/232015521/files/doc_news/2022/11/2022.11.30_-SENS_FDA-ODD-OTOF_ENG.pdf 

2. Sensorion announces US FDA grants Rare Pediatric Disease Designation to OTOF-GT for the treatment of otoferlin gene-mediated hearing loss. News release. Sensorion. November 7, 2022. https://s27.q4cdn.com/232015521/files/doc_news/2022/11/2022.11.07_Sensorion_-Rare-Pediatric-Disease-Designation_Eng.pdf 

3. Decibel therapeutics receives FDA clearance of IND application for DB-OTO, a gene therapy product candidate designed to provide hearing to individuals with otoferlin-related hearing loss. News release. Decibel Therapeutics. October 17, 2022. https://ir.decibeltx.com/news-releases/news-release-details/decibel-therapeutics-receives-fda-clearance-ind-application-db

4. Decibel Therapeutics announces submission of investigational new drug (IND) application for lead gene therapy candidate DB-OTO. News release. Decibel Therapeutics. September 20, 2022. https://www.globenewswire.com/news-release/2022/09/20/2519051/0/en/Decibel-Therapeutics-Announces-Submission-of-Investigational-New-Drug-IND-Application-for-Lead-Gene-Therapy-Candidate-DB-OTO.html 

5. Decibel therapeutics reports third quarter 2022 financial results and corporate update. News release. Decibel Therapeutics. November 9, 2022. https://ir.decibeltx.com/news-releases/news-release-details/decibel-therapeutics-reports-third-quarter-2022-financial 

6. Akouos receives FDA clearance of its IND application for AK-OTOF, a gene therapy intended for the treatment of otof-mediated hearing loss. News release. Akouos, Inc. September 13, 2022. https://investors.akouos.com/news-releases/news-release-details/akouos-receives-fda-clearance-its-ind-application-ak-otof-gene 

7. Akouos reports third quarter 2022 financial results and provides business highlights. News release. Akouos, Inc. November 14, 2022. https://investors.akouos.com/news-releases/news-release-details/akouos-reports-third-quarter-2022-financial-results-and-provides 

8. Lilly and Akouos announce expiration of Akouos tender offer. News release. Eli Lilly and Company. November 30, 2022. https://investor.lilly.com/news-releases/news-release-details/lilly-and-akouos-announce-expiration-akouos-tender-offer