Akouos' Hearing Loss Gene Therapy Poised To Receive Orphan Drug Tag In Europe


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  • The European Medicines Agency (EMA) Committee for Orphan Medicinal Products has issued a positive opinion on Akouos Inc's (NASDAQ:AKUS) application for orphan drug designation for AK-OTOF.
  • AK-OTOF is a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. 
  • The FDA previously granted AK-OTOF Orphan Drug Designation and Rare Pediatric Disease Designation for this same indication.
  • Price Action: AKUS shares are down 4.30% at $11.80 during the market session on the last check Wednesday.
  • Related content: Benzinga's Full FDA Calendar.

Crypto Whales Are Loading Up — Are You?

New research shows the biggest crypto buyers are back. And this time? They could hold for the possibility that Bitcoin will surpass $100,000 in 2024. You don’t want to miss the next massive crypto bull run like we saw in 2020 and 2021. To know exactly what’s going on and what to buy… Get Access To Benzinga’s Best Crypto Research and Investments For Only $1.


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Posted In: BiotechNewsHealth CareSmall CapFDAMoversTrading IdeasGeneralBriefsEuropean Medicines Agency (EMA)gene therapyOrphan Drug Designation