2023-03-30 08:33:19 ET
- The European Commission (EC) granted orphan drug designation to Decibel Therapeutics' ( NASDAQ: DBTX ) gene therapy DB-OTO to treat profound, congenital hearing loss caused by mutations of the otoferlin gene.
- The European Medicines Agency's (EMA) Committee on Orphan Medicinal Products (COMP) had recommended granting the orphan drug status to Decibel's lead gene therapy and this opinion was adopted by the EC.
- "Decibel has generated compelling preclinical data showing DB-OTO’s potential, and we are on track to initiate CHORD, our global Phase 1/2 clinical trial of DB-OTO, in the first half of this year," said Decibel CEO Laurence Reid.
- The company noted that currently, there is no approved pharmacologic therapy for hearing loss caused by genetic mutations of otoferlin.
- The orphan drug status in the EU is for therapies aimed at treating or preventing diseases which affect fewer than five in 10,000 people in the EU. The designation provides incentives, including 10 years' of market exclusivity for the drug, if approved.
- DB-OTO previously received orphan drug and rare pediatric disease designations in the U.S.
For further details see:
Decibel's gene therapy for hearing loss gets orphan drug tag in EU