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

Rhea-AI Summary

Decibel Therapeutics (Nasdaq: DBTX) has received Orphan Drug and Rare Pediatric Disease Designations from the FDA for its lead gene therapy candidate, DB-OTO, aimed at treating otoferlin-related congenital hearing loss. These designations validate the urgent need for innovative treatments in this area and provide Decibel with potential benefits like market exclusivity and tax credits. DB-OTO, developed in collaboration with Regeneron Pharmaceuticals, is designed to restore hearing in patients with profound loss due to otoferlin gene mutations and is expected to enter clinical trials in 2022.

Positive

• Orphan Drug Designation and Rare Pediatric Disease Designation granted by FDA enhance prospects for DB-OTO.
• Potential benefits include up to seven years of market exclusivity and tax credits for clinical development.
• Preclinical studies indicate promising results for restoring hearing in children with otoferlin mutations.
• Collaboration with Regeneron Pharmaceuticals may strengthen development capabilities.

Negative

• None.

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 individuals 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 DB-OTO
DB-OTO is a dual-vector adeno-associated virus (AAV) investigational gene therapy product candidate designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene. The program, developed in collaboration with Regeneron Pharmaceuticals, uses a proprietary, cell-selective promoter to precisely control gene expression in cochlear hair cells. DB-OTO is in preclinical studies, and Decibel expects to initiate clinical testing in 2022.

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 program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit www.decibeltx.com or follow us on Twitter. 

Investor Contact:
Julie Seidel
Stern Investor Relations, Inc.
julie.seidel@sternir.com
212-362-1200

Media Contact:
Chris Railey
Ten Bridge Communications
Chris@tenbridgecommunications.com
617-834-0936 

FAQ

What designations has Decibel Therapeutics received for DB-OTO?

Decibel Therapeutics has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for DB-OTO.

What is DB-OTO and its target patient population?

DB-OTO is a gene therapy product designed to treat congenital hearing loss caused by mutations in the otoferlin gene, primarily affecting children.

When is Decibel Therapeutics planning to initiate clinical trials for DB-OTO?

Decibel Therapeutics plans to initiate Phase 1/2 clinical trials of DB-OTO in pediatric patients in 2022.

What are the potential benefits of the Orphan Drug Designation for DB-OTO?

The Orphan Drug Designation may provide Decibel with market exclusivity for up to seven years, tax credits, and support in the drug development process.

What is the collaboration between Decibel Therapeutics and Regeneron Pharmaceuticals?

Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals, which may enhance the capabilities of the therapy's development.