Decibel Therapeutics Receives Orphan Drug and Rare Pediatric Disease Designations for DB-OTO for the Treatment of Otoferlin-Related Congenital Hearing Loss

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.

To Read the Complete Article at BioSpace Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Stock image by @tatsianam a from Depositphotos