Decibel Therapeutics Receives European Orphan Drug Designation for Lead Gene Therapy Candidate DB-OTO
March 30 2023 - 7:00AM
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 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 TherapeuticsDecibel 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.
Forward-Looking StatementsThis press release
contains forward-looking statements that involve substantial risks
and uncertainties. All statements, other than statements of
historical facts, contained in this press release, including
statements regarding Decibel’s strategy, future operations,
prospects, plans, objectives of management, the therapeutic
potential for Decibel’s product candidates and preclinical
programs, the potential benefits of orphan drug designation in the
European Union, the expected timeline for initiating clinical
trials and expectations regarding the translation of preclinical
findings to human disease constitute forward-looking statements
within the meaning of The Private Securities Litigation Reform Act
of 1995. The words “anticipate,” “believe,” “continue,” “could,”
“estimate,” “expect,” “intend,” “may,” “might,” “objective,”
“ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or
“would,” or the negative of these terms, or other comparable
terminology are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Decibel may not actually achieve the plans,
intentions or expectations disclosed in these forward-looking
statements, and you should not place undue reliance on these
forward-looking statements. Actual results or events could differ
materially from the plans, intentions and expectations disclosed in
these forward-looking statements as a result of various important
factors, including: uncertainties inherent in the identification
and development of product candidates, including the timing of and
Decibel’s ability to obtain approval to initiate clinical
development of its program candidates, whether results from
preclinical studies will be predictive of the results of later
preclinical studies and clinical trials, whether Decibel’s cash
resources are sufficient to fund its foreseeable and unforeseeable
operating expenses and capital expenditure requirements,
uncertainties related to the impact of the COVID-19 pandemic on
Decibel’s business and operations, as well as the risks and
uncertainties identified in Decibel’s filings with the Securities
and Exchange Commission (SEC), including those risks detailed under
the caption “Risk Factors” in Decibel’s Annual Report on Form 10-K
for the fiscal year ended December 31, 2022 and in other filings
Decibel may make with the SEC. In addition, the forward-looking
statements included in this press release represent Decibel’s views
as of the date of this press release. Decibel anticipates that
subsequent events and developments will cause its views to change.
However, while Decibel may elect to update these forward-looking
statements at some point in the future, it specifically disclaims
any obligation to do so. These forward-looking statements should
not be relied upon as representing Decibel’s views as of any date
subsequent to the date of this press release.
Investor Contact:Julie SeidelStern Investor
Relations, Inc.julie.seidel@sternir.com212-362-1200
Media Contact:Chris RaileyTen Bridge
CommunicationsChris@tenbridgecommunications.com617-834-0936
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