Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III
July 27 2021 - 8:00AM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
company focused on the development and commercialization of novel
products for serious rare and ultra-rare genetic diseases, today
announced that the U.S. Food and Drug Administration (FDA) and the
European Commission have granted Orphan Drug Designation for UX053
for the treatment of Glycogen Storage Disease Type III (GSDIII), a
metabolic disease that affects more than 10,000 patients in the
developed world. Enrollment in a Phase 1/2 clinical trial
evaluating the safety, tolerability, and efficacy of UX053 in
adults with GSDIII is expected to begin in the second half of 2021.
About Orphan Drug Designation
The FDA Orphan Drug Designation program provides orphan status
to drugs and biologics that are intended for the safe and effective
treatment, diagnosis, or prevention of rare diseases that affect
fewer than 200,000 people in the U.S. Among the benefits of orphan
designation in the U.S. are seven years of market exclusivity
following FDA approval, waiver or partial payment of application
fees, and tax credits for clinical testing expenses conducted after
orphan designation is received.
The European Commission grants orphan drug designation for
medicinal products intended to treat life-threatening or
chronically debilitating conditions that affect fewer than five in
10,000 people in the European Union and when no satisfactory method
of diagnosis, prevention or treatment of the condition can be
authorized. The designation provides certain benefits and
incentives in the EU, including protocol assistance, fee
reductions, and ten years of market exclusivity once the medicine
is on the market.
About Glycogen Storage Disease Type III
Glycogen Storage Disease Type III, or GSDIII, a disease caused
by a glycogen debranching enzyme (AGL) deficiency that results in
glycogen accumulation in the liver and muscle. GSDIII can cause
hepatomegaly, hypoglycemia, hyperlipidemia, some progressive liver
cirrhosis, and skeletal and cardiac muscle disease. There are no
approved treatment options for GSDIII and the current standard of
care is a strict diet, including frequent doses of cornstarch, to
reduce the risk of hypoglycemia. GSDIII affects more than 10,000
patients in the developed world.
About UX053
UX053 is an investigational mRNA-based biologic therapy encoding
full-length, glycogen debranching enzyme encapsulated in a lipid
nanoparticle (LNP) designed to provide the deficient protein in
GSDIII. Ultragenyx is developing UX053 and a number of other mRNA
therapies in the preclinical stage for undisclosed indications
pursuant to its collaboration with Arcturus Therapeutics, a
clinical-stage messenger RNA medicines company
About Ultragenyx Pharmaceutical Inc.Ultragenyx
is a biopharmaceutical company committed to bringing novel products
to patients for the treatment of serious rare and ultra-rare
genetic diseases. The company has built a diverse portfolio of
approved therapies and product candidates aimed at addressing
diseases with high unmet medical need and clear biology for
treatment, for which there are typically no approved therapies
treating the underlying disease.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx’s strategy is predicated upon time- and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's
website at: www.ultragenyx.com.
Forward-Looking Statements
Except for the historical information contained herein, the
matters set forth in this press release, including statements
related to Ultragenyx's expectations and projections regarding its
future operating results and financial performance, anticipated
cost or expense reductions, the timing, progress and plans for its
clinical programs and clinical studies, future regulatory
interactions, and the components and timing of regulatory
submissions are forward-looking statements within the meaning of
the "safe harbor" provisions of the Private Securities Litigation
Reform Act of 1995. Such forward-looking statements involve
substantial risks and uncertainties that could cause our clinical
development programs, collaboration with third parties, future
results, performance or achievements to differ significantly from
those expressed or implied by the forward-looking statements. Such
risks and uncertainties include, among others, the effects from the
COVID-19 pandemic on the company’s clinical activities, business
and operating results, risks related to reliance on third party
partners to conduct certain activities on the company’s behalf,
uncertainty and potential delays related to clinical drug
development, smaller than anticipated market opportunities for the
company’s products and product candidates, manufacturing risks,
competition from other therapies or products, and other matters
that could affect sufficiency of existing cash, cash equivalents
and short-term investments to fund operations, the company’s future
operating results and financial performance, the timing of clinical
trial activities and reporting results from same, and the
availability or commercial potential of Ultragenyx’s products and
drug candidates. Ultragenyx undertakes no obligation to update or
revise any forward-looking statements. For a further description of
the risks and uncertainties that could cause actual results to
differ from those expressed in these forward-looking statements, as
well as risks relating to the business of Ultragenyx in general,
see Ultragenyx's Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission on May 5, 2021, and its
subsequent periodic reports filed with the Securities and Exchange
Commission.
Contact Ultragenyx Pharmaceutical Inc.InvestorsJoshua
Higa415-660-0951
Media Carolyn Wang415-225-5050
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