Protara Therapeutics Receives Rare Pediatric Disease Designation for TARA-002 for the Treatment of Lymphatic Malformations
July 28 2020 - 6:00AM
Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical stage company
developing treatments for rare and specialty diseases with
significant unmet needs, today announced that the U.S. Food and
Drug Administration (FDA) has granted Rare Pediatric Disease
designation for TARA-002 for the treatment of Lymphatic
Malformations (LMs). TARA-002 is an investigational
cell-based therapy based on the broad immunopotentiator OK-432,
which is approved in Japan and Taiwan for the treatment of LMs. LMs
are rare, typically congenital, malformations of the lymphatic
vasculature.
“Receipt of Rare Pediatric Disease designation from the FDA for
TARA-002 in LMs further underscores the significant unmet medical
need for the children that are affected by this rare and serious
disease,” said Jesse Shefferman, Chief Executive Officer
of Protara Therapeutics. “We are encouraged about the
potential of TARA-002 to enhance the treatment paradigm of LMs for
this underserved population, and we look forward to working with
the agency to determine next steps for the program in the coming
months.”
About FDA Rare Pediatric Disease
Designation
The FDA grants Rare Pediatric Disease
designation for serious diseases that primarily affect children
ages 18 years or younger and fewer than 200,000 persons in the
United States. Under the FDA's Rare Pediatric Disease
Priority Review Voucher program, a sponsor who receives an approval
of a new drug application or biologics license application for a
product for the prevention or treatment of a rare pediatric disease
may be eligible for a voucher, which can be redeemed to obtain
priority review for any subsequent marketing application or may be
sold or transferred.
About TARA-002 for Lymphatic
Malformations
TARA-002, Protara’s lead product candidate, is
an investigational cell-based therapy based on the broad
immunopotentiator OK-432. OK-432 is approved in Japan and Taiwan
for lymphatic malformations (LMs), which are rare, congenital
malformations of lymphatic vessels resulting in the failure of
these structures to connect or drain into the venous system. In a
randomized, Phase 2 clinical trial of OK-432 in LMs, 86% of
patients treated with OK-432 (>90% pediatric) experienced a
complete or substantial response. Long-term control of LMs was
favorable, with more than 90% of patients treated with OK-432
having no regrowth three years following treatment.
TARA-002 has been granted Rare Pediatric Disease
designation by the U.S. Food and Drug Administration for the
treatment of LMs.
About Protara Therapeutics,
Inc.
Protara is committed to identifying and
advancing transformative therapies for people with rare and
specialty diseases who have limited treatment options. Protara’s
portfolio includes its lead program, TARA-002, an investigational
cell-based therapy being developed for the treatment of lymphatic
malformations, and IV Choline Chloride, an investigational
phospholipid substrate replacement therapy for the treatment of
IFALD. For more information, visit www.protaratx.com
Forward-Looking Statements
Statements contained in this press release
regarding matters that are not historical facts are
"forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995. Because such statements
are subject to risks and uncertainties, actual results may differ
materially from those expressed or implied by such forward-looking
statements. Such statements include, but are not limited to,
statements regarding the potential that Protara will receive a
priority review voucher from the FDA. Risks that contribute to the
uncertain nature of the forward-looking statements include:
uncertainties related to Protara’s development programs, including
the initiation and completion of non-clinical studies and clinical
trials and the timing of required filings with the FDA and other
regulatory agencies; and uncertainties related to the actual
impacts and length of such impacts caused by the COVID-19 pandemic.
These and other risks and uncertainties are described more fully
under the caption "Risk Factors" and elsewhere in Protara's filings
and reports with the United States Securities and Exchange
Commission. All forward-looking statements contained in this press
release speak only as of the date on which they were made. Protara
undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on
which they were made.
Company Contact:
Blaine Davis Protara Therapeutics Blaine.Davis@protaratx.com
646-844-0337
Protara Therapeutics (NASDAQ:TARA)
Historical Stock Chart
From Sep 2024 to Oct 2024
Protara Therapeutics (NASDAQ:TARA)
Historical Stock Chart
From Oct 2023 to Oct 2024