Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation and Rare Pediatric Disease Designation for the company’s lead gene therapy product candidate, DB-OTO, for the treatment of patients with otoferlin-related congenital hearing loss.
“We are pleased to receive these important designations from the FDA, which support our conviction that innovative treatments for congenital hearing loss are urgently needed,” said Heather Wolff, Vice President, Clinical Development Operations of Decibel. “Preclinical studies support the potential of DB-OTO to provide hearing to children born with profound hearing loss due to a mutation of the otoferlin gene. We are looking forward to initiating a Phase 1/2 clinical trial of DB-OTO in pediatric patients in 2022.”
Orphan Drug Designation is granted to drugs and biologics intended for the treatment, diagnosis or prevention of rare diseases, or conditions affecting fewer than 200,000 people in the United States. The designation affords Decibel the potential for certain benefits, including up to seven years of post-approval market exclusivity, assistance in the drug development process, tax credits for clinical development and exemptions from certain FDA fees.
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