Decibel Therapeutics Receives Orphan Drug and Rare Pediatric Disease Designations for DB-OTO for the Treatment of Otoferlin-Related Congenital Hearing Loss
BOSTON, Sept. 09, 2021 (GLOBE NEWSWIRE) -- 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.
Rare Pediatric Disease Designation is granted by the FDA to encourage development of treatments for serious or life-threatening rare diseases in which the disease manifestations primarily affect inividuals aged from birth to 18 years. Under the Priority Review Voucher program, and subject to FDA approval of DB-OTO for the treatment of otoferlin-related hearing loss, Decibel may be eligible to receive one priority review voucher, which could then be redeemed to receive priority review for a subsequent marketing application for a different product or sold or transferred to another sponsor.
About Decibel Therapeutics
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