Protara Therapeutics Receives Fast Track Designation from U.S. FDA for Intravenous Choline Chloride for the Treatment of Inte...
May 26 2020 - 7:30AM
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 Fast Track designation to
Intravenous (IV) Choline Chloride for the treatment of intestinal
failure-associated liver disease (IFALD). IV Choline Chloride is
the Company’s Phase 3-ready investigational phospholipid substrate
replacement therapy for patients receiving parenteral nutrition
(PN) who have IFALD.
“Receiving Fast Track designation from the FDA
further supports the potential for IV Choline Chloride to serve as
the much-needed first approved therapy for IFALD patients,” said
Jesse Shefferman, Chief Executive Officer of Protara
Therapeutics. “We look forward to collaborating with FDA and
utilizing the benefits associated with Fast Track designation to
make this important therapy available for patients with IFALD.”
Data from the randomized, controlled, Phase 2
trial in IFALD patients dependent on PN demonstrated that treatment
with IV Choline Chloride resulted in normalization of plasma-free
choline concentrations, reversal of hepatic steatosis, and a
clinically meaningful and statistically significant improvement in
cholestasis.
Protara held an end of Phase 2 meeting with the
FDA in late 2018 and received the FDA’s support to advance IV
Choline Chloride into a registration-enabling study for the
treatment of IFALD.
About FDA Fast Track
Designation
The FDA’s Fast Track program facilitates the
development and expedites the review of drugs that treat serious
conditions and have the potential to address an unmet medical need.
Programs with Fast Track designation may benefit from early and
frequent interactions with the FDA over the course of drug
development. In addition, the Fast Track designation program allows
for the eligibility for accelerated approval and priority review if
relevant criteria are met, and enables a company to submit
individual sections of a New Drug Application (NDA) for review on a
rolling-submission basis.
About Intravenous (IV) Choline Chloride
for IFALD
Intravenous (IV) Choline Chloride is a Phase
3-ready investigational phospholipid substrate replacement therapy
initially in development for patients receiving parenteral
nutrition (PN) who have intestinal failure-associated liver disease
(IFALD). Protara believes that IV Choline Chloride has the
potential to be the first approved therapy for IFALD. Choline is a
known important substrate for phospholipids that are critical for
healthy liver function, yet currently available PN formulations do
not contain sufficient amounts of choline. IV Choline Chloride has
been granted Orphan Drug Designation for the treatment of IFALD and
prevention of choline deficiency in PN patients. IV Choline
Chloride has also been granted Fast Track Designation for the
treatment of IFALD.
About IFALD
IFALD is uniquely characterized by the presence
of both steatosis (toxic fat accumulation in liver cells) and
cholestasis (damage to the biliary system in the liver) in patients
who are chronic (greater than six months) PN users.
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 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 Protara’s business strategy, Protara’s
development plans for IV Choline Chloride and Protara’s plans for
interactions with the FDA for this product candidate. 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; uncertainties related to the
actual impacts and length of such impacts caused by the COVID-19
pandemic; having to use cash in ways or on timing other than
expected; and the impact of market volatility on cash reserves.
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
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