Travere Therapeutics Announces European Commission Has Granted Orphan Designation to Sparsentan for the Treatment of IgA Neph...
February 18 2021 - 4:30PM
Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced the
European Commission (EC) has granted orphan designation to
sparsentan for the treatment of IgA nephropathy (IgAN), a rare
kidney disorder and a leading cause of end-stage kidney disease
(ESKD). Sparsentan is an investigational product candidate
currently being evaluated in a pivotal Phase 3 clinical study for
the treatment of IgAN, as well as a pivotal Phase 3 clinical study
for the treatment of focal segmental glomerulosclerosis (FSGS). The
Company recently reported that the ongoing DUPLEX Study of
sparsentan in FSGS achieved its pre-specified interim proteinuria
endpoint and that preliminary results from the interim analysis
suggest that to date in the study, sparsentan has been generally
well-tolerated and has shown a comparable safety profile to
irbesartan. Topline data from the interim proteinuria assessment in
the ongoing PROTECT Study of sparsentan in IgAN remain on track to
be reported in the third quarter of 2021.
“The limited and non-specific therapeutic
strategies used for IgAN today are often associated with long-term
tolerability challenges, and for many are not enough to slow the
progression to ESKD,” said Noah Rosenberg, M.D., chief medical
officer of Travere Therapeutics. “We are pleased to receive orphan
designation in Europe which further supports our goal of ultimately
delivering sparsentan as a potential new treatment standard for
IgAN.”
Orphan designation from the EC provides
incentives for companies to develop medicines intended for the
treatment, prevention or diagnosis of a disease that is
life-threatening or chronically debilitating and where no
satisfactory treatment is currently authorized. The prevalence of
the condition must not exceed more than five in 10,000 people in
the European Union (EU). In addition to being eligible for a
10-year period of marketing exclusivity in the EU upon product
approval, orphan designation provides fee waivers, protocol
assistance, and eligibility for marketing authorization under the
centralized procedure granting approval in all EU countries.
Travere Therapeutics was previously granted Orphan Drug designation
for IgAN in the U.S., and it also holds Orphan Drug designation in
the U.S. and orphan designation in Europe for sparsentan for the
treatment of FSGS.
About Sparsentan
Sparsentan is a novel investigational product
candidate, that functions as a high affinity dual-acting antagonist
of both the endothelin type A and angiotensin II type 1 receptors,
in a single molecule. Pre-clinical data have shown that blockade of
both pathways in forms of rare chronic kidney disease, reduces
proteinuria, protects podocytes and prevents glomerulosclerosis and
mesangial cell proliferation.
Sparsentan is currently being evaluated in the
pivotal Phase 3 PROTECT Study for the treatment of IgAN and the
pivotal Phase 3 DUPLEX Study for the treatment of FSGS. In February
of 2021, the Company reported that the ongoing DUPLEX Study of
sparsentan in FSGS achieved its pre-specified interim proteinuria
endpoint and that preliminary results from the interim analysis
suggest that to date in the study, sparsentan has been generally
well-tolerated and has shown a comparable safety profile to
irbesartan. In the Phase 2 DUET Study of sparsentan in FSGS, the
combined treatment group met its primary efficacy endpoint,
demonstrating a greater than two-fold reduction in proteinuria
compared to irbesartan, and was generally well tolerated after the
eight-week, double-blind treatment period. Irbesartan is part of a
class of drugs used to manage FSGS and IgAN in the absence of an
approved pharmacologic treatment. If approved for both indications,
sparsentan could potentially be the first medicine approved for
both FSGS and IgAN.
About Travere Therapeutics
At Travere Therapeutics we are in rare for life.
We are a biopharmaceutical company that comes together every day to
help patients, families and caregivers of all backgrounds as they
navigate life with a rare disease. On this path, we know the need
for treatment options is urgent – that is why our global team works
with the rare disease community to identify, develop and deliver
life-changing therapies. In pursuit of this mission, we
continuously seek to understand the diverse perspectives of rare
patients and to courageously forge new paths to make a difference
in their lives and provide hope – today and tomorrow. For more
information, visit travere.com
Forward Looking Statements
This press release contains "forward-looking
statements" as that term is defined in the Private Securities
Litigation Reform Act of 1995. Without limiting the foregoing,
these statements are often identified by the words "may", "might",
"believes", "thinks", "anticipates", "plans", "expects", "intends"
or similar expressions. In addition, expressions of our strategies,
intentions or plans are also forward-looking statements. Such
forward-looking statements include, but are not limited to,
references to the Company’s current expectations around timelines
for reporting top-line data from the proteinuria endpoint in the
PROTECT study, the goal of ultimately delivering sparsentan as a
potential new treatment standard for IgAN. Such forward-looking
statements are based on current expectations and involve inherent
risks and uncertainties, including factors that could delay, divert
or change any of them, and could cause actual outcomes and results
to differ materially from current expectations. No forward-looking
statement can be guaranteed. Among the factors that could cause
actual results to differ materially from those indicated in the
forward-looking statements are risks and uncertainties associated
with the Company’s business and finances in general, success of its
commercial products as well as risks and uncertainties associated
with the Company's preclinical and clinical stage pipeline.
Specifically, the Company faces risks associated with market
acceptance of its commercial products including efficacy, safety,
price, reimbursement and benefit over competing therapies. The
risks and uncertainties the Company faces with respect to its
preclinical and clinical stage pipeline include risk that the
Company's clinical candidates will not be found to be safe or
effective and that current clinical trials will not proceed as
planned. Specifically, the Company faces the risk that the Phase 3
clinical trial of sparsentan in FSGS will not demonstrate that
sparsentan is safe or effective or serve as a basis for accelerated
approval of sparsentan as planned; risk that the Phase 3 clinical
trial of sparsentan in IgAN will not demonstrate that sparsentan is
safe or effective or serve as the basis for accelerated approval of
sparsentan as planned; and for each of its development programs,
risk associated with enrollment of clinical trials for rare
diseases and risk that ongoing clinical trials may not proceed on
expected timelines or may be delayed for safety, regulatory or
other reasons and risk that the product candidates will not be
approved for efficacy, safety, regulatory or other reasons. The
Company faces risk that it will be unable to raise additional
funding that may be required to complete development of any or all
of its product candidates; risk relating to the Company's
dependence on contractors for clinical drug supply and commercial
manufacturing; uncertainties relating to patent protection and
exclusivity periods and intellectual property rights of third
parties; risks associated with regulatory interactions; and risks
and uncertainties relating to competitive products, including
potential generic competition with certain of the Company’s
products, and technological changes that may limit demand for the
Company's products. The Company faces additional risks associated
with the potential impacts the COVID-19 pandemic may have on its
business, including, but not limited to (i) the Company’s ability
to continue its ongoing development activities and clinical trials,
(ii) the timing of such clinical trials and the release of data
from those trials, (iii) the Company’s and its suppliers’ ability
to successfully manufacture its commercial products and product
candidates, and (iv) the market for and sales of its commercial
products. You are cautioned not to place undue reliance on these
forward-looking statements as there are important factors that
could cause actual results to differ materially from those in
forward-looking statements, many of which are beyond our control.
The Company undertakes no obligation to publicly update any
forward-looking statement, whether as a result of new information,
future events, or otherwise. Investors are referred to the full
discussion of risks and uncertainties as included in the Company's
most recent Form 10-Q, Form 10-K and other filings with the
Securities and Exchange Commission.
Contact:Chris Cline, CFASenior Vice President,
Investor Relations & Corporate
Communications888-969-7879IR@travere.com
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