Boston — Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, announced that the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) has issued a positive opinion on orphan drug designation for DB-OTO, Decibel’s lead gene therapy product candidate designed to provide durable, high quality, physiological hearing to individuals with profound, congenital hearing loss caused by mutations of the otoferlin gene. This opinion was adopted by the European Commission (EC).
“We are pleased to receive this important designation from the EC, which supports our conviction that innovative treatments for congenital hearing loss are urgently needed,” said Laurence Reid, Ph.D., Chief Executive Officer at Decibel. “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.”
Orphan drug designation is granted by the EC for medicines in development to treat rare conditions affecting no more than five in 10,000 people in the European Union, provided there is no other satisfactory treatment option or the medicine can be of significant benefit to those affected by a specific condition. Medicines that are granted orphan drug designation by the EC qualify for financial and regulatory incentives including protocol assistance, possible exemptions or reductions in certain regulatory fees, and, if approved for marketing, ten years of market exclusivity in the European Union.
DB-OTO received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration in 2021. Currently, there are no approved pharmacologic treatment options for people with hearing loss caused by genetic mutations of otoferlin.
About Decibel Therapeutics
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel’s pipeline, including its lead gene therapy product candidate, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of creating a world of connection for people with hearing and balance disorders. For more information about Decibel Therapeutics, please visit www.decibeltx.com or follow us on Twitter.