Travere Therapeutics, Inc., (NASDAQ: TVTX) and CSL Vifor today
announced that the European Medicines Agency’s (EMA) CHMP has
recommended approval of sparsentan for the treatment of adults with
primary IgA nephropathy (IgAN) with a urine protein excretion
>1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g).
IgAN is a rare kidney disease and a leading cause of kidney
failure. The CHMP opinion provides the basis for the European
Commission’s final decision regarding CMA for sparsentan. If
approved in Europe, sparsentan will be the first
non-immunosuppressive, single-molecule, dual endothelin angiotensin
receptor antagonist for the treatment of IgAN.
“The positive recommendation from the CHMP
represents a significant advancement toward the delivery of new
treatment options for people living with IgAN in Europe, who face
the risk of progression to kidney failure and who currently have no
approved non-immunosuppressive treatment options. The PROTECT Study
is the only head-to-head study in IgAN against a maximally labeled
dose of irbesartan, a current standard of care. The study
demonstrated treatment with sparsentan resulted in a rapid and
sustained reduction in proteinuria and has the potential to
preserve kidney function and significantly delay time to kidney
failure compared to an active comparator, suggesting long-term
benefits in IgAN,” said Eric Dube, Ph.D., president and chief
executive officer of Travere Therapeutics. “Together with CSL
Vifor, we look forward to the European Commission’s decision in the
second quarter of 2024.”
“The positive CHMP opinion for sparsentan is one
step closer to bringing this treatment option to patients in Europe
with IgAN, a rare and serious condition that can cause kidney
disease,” said Emmanuelle Lecomte Brisset, senior vice president
and head of global regulatory affairs at CSL. “We look forward to
the European Commission decision and to continuing to advance CSL’s
promise to provide innovative treatments for patients with kidney
disease.”
The positive CHMP opinion is based on results
from the pivotal Phase 3 PROTECT Study of sparsentan in IgAN.
In August 2022, Travere Therapeutics and CSL
Vifor announced they had submitted a Marketing Authorization
Application (MAA) for CMA to the EMA. The European Commission
previously granted Orphan Medicinal Product Designation to
sparsentan for the treatment of IgAN.
If approved, sparsentan would receive a CMA in
all member states of the European Union, as well as in Iceland,
Liechtenstein and Norway. Sparsentan is currently marketed in the
U.S. and granted accelerated approval by the U.S. Food and Drug
Administration under the brand name FILSPARI® based on reduction in
proteinuria.
In 2021, Travere Therapeutics granted CSL Vifor
exclusive commercialization rights for sparsentan in Europe,
Australia and New Zealand.
About IgA Nephropathy
(IgAN)
IgAN, also called Berger’s disease, is a rare
progressive kidney disease characterized by the buildup of
immunoglobulin A (IgA), a protein that helps the body fight
infections, in the kidneys. The deposits of IgA cause a breakdown
of the normal filtering mechanisms in the kidney, leading to blood
in the urine (hematuria), protein in the urine (proteinuria) and a
progressive loss of kidney function. Other symptoms of IgAN may
include swelling (edema) and high blood pressure.
IgAN is the most common type of primary
glomerular disease worldwide and a leading cause of kidney failure.
IgAN is estimated to affect up to 250,000 people in the licensed
territories.
About the PROTECT Study
The PROTECT Study is one of the largest
interventional studies to date in IgA nephropathy (IgAN) and the
only head-to-head trial in this rare kidney disease. It is a
global, randomized, multicenter, double-blind, parallel-arm,
active-controlled clinical trial evaluating the safety and efficacy
of 400 mg of sparsentan, compared to 300 mg of irbesartan, in 404
patients ages 18 years and up with IgAN and persistent proteinuria
despite receiving at least 50% of max label dose and maximally
tolerated ACE or ARB therapy.
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
About CSL Vifor
CSL Vifor is a global partner of choice for
pharmaceuticals and innovative, leading therapies in iron
deficiency and nephrology. We specialize in strategic global
partnering, in-licensing and developing, manufacturing and
marketing pharmaceutical products for precision healthcare, aiming
to help patients around the world lead better, healthier lives.
Headquartered in St. Gallen, Switzerland, CSL Vifor also includes
the joint company Vifor Fresenius Medical Care Renal Pharma (with
Fresenius Medical Care).
The parent company, CSL (ASX: CSL; USOTC:
CSLLY), headquartered in Melbourne, Australia, employs 32,000
people and delivers its lifesaving therapies to people in more than
100 countries. For more information about CSL Vifor visit,
www.cslvifor.com.
FILSPARI® (sparsentan) U.S.
Indication
FILSPARI is an endothelin and
angiotensin II receptor antagonist indicated to reduce proteinuria
in adults with primary immunoglobulin A nephropathy (IgAN) at risk
of rapid disease progression, generally a UPCR ≥1.5
g/g.
This indication is granted under
accelerated approval based on reduction in proteinuria. It has not
been established whether FILSPARI slows kidney function decline in
patients with IgAN. Continued approval for this indication may be
contingent upon verification and description of clinical benefit in
a confirmatory clinical trial.
FILSPARI® (sparsentan) Important Safety
Information
BOXED WARNING: HEPATOTOXICITY AND
EMBRYO-FETAL TOXICITYBecause of the risks of
hepatotoxicity and birth defects, FILSPARI is available only
through a restricted program called the FILSPARI REMS. Under the
FILSPARI REMS, prescribers, patients and pharmacies must enroll in
the program.
HepatotoxicitySome
Endothelin Receptor Antagonists (ERAs) have caused elevations of
aminotransferases, hepatotoxicity, and liver failure. In clinical
studies, elevations in aminotransferases (ALT or AST) of at least
3-times the Upper Limit of Normal (ULN) have been observed in up to
2.5% of FILSPARI-treated patients, including cases confirmed with
rechallenge.
Measure transaminases and bilirubin
before initiating treatment and monthly for the first 12 months,
and then every 3 months during treatment. Interrupt treatment and
closely monitor patients who develop aminotransferase elevations
more than 3x ULN.
FILSPARI should generally be avoided in
patients with elevated aminotransferases (>3x ULN) at baseline
because monitoring for hepatotoxicity may be more difficult and
these patients may be at increased risk for serious
hepatotoxicity.
Embryo-Fetal
ToxicityFILSPARI can cause major birth defects if
used by pregnant patients based on animal data. Therefore,
pregnancy testing is required before the initiation of treatment,
during treatment and one month after discontinuation of treatment
with FILSPARI. Patients who can become pregnant must use effective
contraception before the initiation of treatment, during treatment,
and for one month after discontinuation of treatment with
FILSPARI.
Contraindications: FILSPARI is
contraindicated in patients who are pregnant. Do not coadminister
FILSPARI with angiotensin receptor blockers (ARBs), ERAs, or
aliskiren.
Warnings and Precautions
Hepatotoxicity: Elevations in ALT or AST of at
least 3-fold ULN have been observed. To reduce the risk of
potential serious hepatotoxicity, measure serum aminotransferase
levels and total bilirubin prior to initiation of treatment,
monthly for the first 12 months, then every 3 months during
treatment.
Advise patients with symptoms suggesting
hepatotoxicity (nausea, vomiting, right upper quadrant pain,
fatigue, anorexia, jaundice, dark urine, fever, or itching) to
immediately stop treatment with FILSPARI and seek medical
attention. If aminotransferase levels are abnormal at any time
during treatment, interrupt FILSPARI and monitor as
recommended.
Consider re-initiation of FILSPARI only when
hepatic enzyme levels and bilirubin return to pretreatment values
and only in patients who have not experienced clinical symptoms of
hepatotoxicity.
Avoid initiation of FILSPARI in patients with
elevated aminotransferases (>3x ULN) prior to drug
initiation.
Embryo-Fetal Toxicity: FILSPARI
can cause fetal harm. Advise patients who can become pregnant of
the potential risk to a fetus. Obtain a pregnancy test and advise
patients who can become pregnant to use effective contraception
prior to, during, and one month after discontinuation of FILSPARI
treatment.
FILSPARI REMS: FILSPARI is
available only through a restricted program under a REMS called the
FILSPARI REMS.Important requirements include:— Prescribers must be
certified with the FILSPARI REMS by enrolling and completing
training.— All patients must enroll in the FILSPARI REMS prior to
initiating treatment and comply with monitoring requirements.—
Pharmacies that dispense FILSPARI must be certified with the
FILSPARI REMS and must dispense only to patients who are authorized
to receive FILSPARI.Further information is available at
www.filsparirems.com or 1-833-513-1325.
Hypotension: There was a
greater incidence of hypotension-associated adverse events, some
serious, including dizziness, in patients treated with FILSPARI
compared to irbesartan. In patients at risk for hypotension,
consider eliminating or adjusting other antihypertensive
medications and maintaining appropriate volume status. If
hypotension develops, consider a dose reduction or dose
interruption of FILSPARI.
Acute Kidney Injury: Monitor
kidney function periodically. Patients whose kidney function may
depend in part on the activity of the renin-angiotensin system
(e.g., patients with renal artery stenosis, chronic kidney disease,
severe congestive heart failure, or volume depletion) may be at
particular risk of developing acute kidney injury on FILSPARI.
Consider withholding or discontinuing therapy in patients who
develop a clinically significant decrease in kidney function while
on FILSPARI.
Hyperkalemia: Monitor serum
potassium periodically and treat appropriately. Patients with
advanced kidney disease, taking concomitant potassium-increasing
drugs (e.g., potassium supplements, potassium-sparing diuretics),
or using potassium-containing salt substitutes are at increased
risk for developing hyperkalemia. Dosage reduction or
discontinuation of FILSPARI may be required.
Fluid Retention: Fluid
retention may occur with ERAs, and has been observed with FILSPARI.
If clinically significant fluid retention develops, after
evaluation, consider modifying the dose of FILSPARI.
Most common adverse reactions (5%) with
FILSPARI are peripheral edema, hypotension (including
orthostatic hypotension), dizziness, hyperkalemia, and anemia.
Drug interactions
- Renin-Angiotensin System
(RAS) Inhibitors and ERAs: Do not coadminister FILSPARI
with angiotensin receptor blockers (ARBs), ERAs, or aliskiren.
- Strong and Moderate CYP3A
Inhibitors: Avoid concomitant use of FILSPARI with strong
CYP3A inhibitors. Monitor blood pressure, serum potassium, edema,
and kidney function regularly when used concomitantly with moderate
CYP3A inhibitors.
- Strong CYP3A
Inducers: Avoid concomitant use with a strong CYP3A
inducer.
- Antacids and Acid Reducing
Agents: Administer FILSPARI 2 hours before or after
administration of antacids. Avoid concomitant use of acid reducing
agents (histamine H2 receptor antagonist and PPI proton pump
inhibitor) with FILSPARI.
- Non-Steroidal
Anti-Inflammatory Agents (NSAIDs), Including Selective
Cyclooxygenase-2 (COX-2) Inhibitors: Monitor for signs of
worsening renal function.
- CYP2B6, 2C9, and 2C19
Substrates: Monitor for efficacy of the concurrently
administered CYP2B6, 2C9, and 2C19 substrates and consider dosage
adjustment in accordance with the Prescribing Information.
- P-gp and BCRP
Substrates: Avoid concomitant use of sensitive substrates
of P-gp and BCRP with FILSPARI.
- Agents Increasing Serum
Potassium: Monitor serum potassium frequently. Concomitant
use of FILSPARI with potassium-sparing diuretics, potassium
supplements, potassium-containing salt substitutes, or other drugs
that raise serum potassium levels may result in hyperkalemia.
Use in specific populations
- Pregnancy / Females and
Males of Reproductive Potential: FILSPARI can cause fetal
harm, including birth defects and fetal death, when administered to
a pregnant patient and is contraindicated during pregnancy.
- Pregnancy Testing /
Contraception: Verify the pregnancy status and effective
method of contraception prior to, during, and one month after
discontinuation of FILSPARI treatment. The patient should contact
their physician immediately for pregnancy testing if onset of
menses is delayed or pregnancy is suspected.
- Lactation: Advise
patients not to breastfeed during treatment with FILSPARI.
- Hepatic
Impairment: Avoid use of FILSPARI in patients with any
hepatic impairment (Child-Pugh class A-C).
Please see Full Prescribing Information
for FILSPARI here.
Forward-Looking StatementsThis
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 “on-track,” “positioned,” “look forward
to,” “will,” “would,” “may,” “might,” “believes,” “anticipates,”
“plans,” “expects,” “intends,” “potential,” 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: statements
regarding the potential conditional marketing authorization of
sparsentan for the treatment of IgAN in the European Union,
Iceland, Liechtenstein and Norway and the anticipated timing
thereof, including the potential timing and outcome of the European
Commission’s decision; and the potential for sparsentan to be the
first non-immunosuppressive, single-molecule, dual endothelin
angiotensin receptor antagonist for the treatment of IgAN in the
EU. 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
regulatory review and approval process, as well as risks and
uncertainties associated with the Company’s business and finances
in general, the success of its commercial products and 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, as well as risks associated with the
successful development and execution of commercial strategies for
such products, including FILSPARI. 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 or
anticipated future clinical trials will not proceed as planned.
Specifically, the Company faces risks related to the timing and
potential outcome of the European Commission’s decision regarding
conditional marketing authorization of sparsentan for IgAN. There
is no guarantee that the European Commission will grant conditional
marketing authorization of sparsentan for IgAN or that regulators
will grant full approval of sparsentan for IgAN. The Company also
faces the 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, including as a result of macroeconomic
conditions; risks 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 current and potential future
generic competition with certain of the Company’s products, and
technological changes that may limit demand for the Company’s
products. The Company also faces additional risks associated with
global and macroeconomic conditions, including health epidemics and
pandemics, including risks related to potential disruptions to
clinical trials, commercialization activity, supply chain, and
manufacturing operations. 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, including under the
heading “Risk Factors”, as included in the Company’s most recent
Form 10-K, Form 10-Q and other filings with the Securities and
Exchange Commission.
Travere Therapeutics Contact:
Media:888-969-7879mediarelations@travere.com |
Investors:888-969-7879IR@travere.com |
CSL Vifor Media ContactThomas
HutterMobile: +41 79 957 96 73Email: media@viforpharma.com |
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