Ipsen confirms U.S. FDA grants priority review for New Drug
Application for elafibranor for the treatment of rare cholestatic
liver disease, PBC
- New Drug Application
granted priority review with PDUFA date set for June 10,
2024
- European Medicines Agency
(EMA) has also validated the Marketing Authorization Application
(MAA) for elafibranor
- Investigational elafibranor
is the first novel second-line treatment for primary biliary
cholangitis (PBC) to be filed in E.U. and U.S. in nearly a
decade
Paris (France), December 07,
2023 – Ipsen (Euronext: IPN; ADR: IPSEY) and
GENFIT (Nasdaq and Euronext: GNFT) today announced that
the U.S. Food and Drug Administration (FDA) has accepted the New
Drug Application (NDA) for investigational elafibranor. An oral,
once-daily dual peroxisome activated receptor alpha/delta (PPAR
α,δ) agonist, investigational elafibranor could potentially be the
first novel second-line treatment for the rare, cholestatic liver
disease, PBC, in nearly a decade. The target FDA PDUFA date under
priority review is June 10, 2024.
The European Medicines Agency (EMA) has also validated Ipsen’s
Marketing Authorization Application (MAA) for elafibranor and the
review of the submission to the EMA’s Committee for Medicinal
Products for Human Use (CHMP) began on 26 October 2023.
Furthermore, a third simultaneous regulatory filing of elafibranor
has been validated for review by the UK Medicines and Healthcare
products Regulatory Agency (MHRA).
“We are delighted to have achieved simultaneous filings for
elafibranor, which is in line with our ambition to be able to bring
a new and much needed medicine to as many people living with PBC as
rapidly as possible,” said Christelle Huguet, EVP and Head
of Research & Development, Ipsen. “This is a condition
where many patients are living with worsening disease and
debilitating symptoms despite being on treatment. Elafibranor, if
approved, has the potential to change the management of this
challenging condition for people living with PBC, offering a new
second line treatment choice, where the number of effective options
are currently limited.”
PBC is a rare, progressive, autoimmune
cholestatic liver disease1 in which bile ducts in the liver are
gradually destroyed.2 The damage to bile ducts can inhibit the
liver’s ability to rid the body of toxins, and can lead to scarring
of liver tissue, known as cirrhosis.1,2,3 Common symptoms of PBC
include fatigue and pruritus (itch), which can be severely
debilitating.4 Untreated, PBC can lead to liver failure, or in some
cases death.1 It primarily affects women, with nine women diagnosed
for every man.3 A significant proportion of people living with PBC
do not benefit from existing therapies.5, 6 7
“These simultaneous regulatory submission acceptances are
another important step in the elafibranor journey. We are pleased
to be partnering with Ipsen, who we know has a good understanding
of the rare-disease regulatory process,” said Pascal
Prigent, Chief Executive Officer of GENFIT. “We know they
share the same goal as GENFIT, to bring a new, much needed
treatment option to people living with PBC as fast as possible; we
look forward to elafibranor’s progress through the regulatory
review processes.”
ABOUT ELAFIBRANOR
Elafibranor is an oral, once-daily, dual
peroxisome activated receptor (PPAR) alpha/delta (α,δ) agonist,
currently under investigation as a treatment for patients with PBC,
a rare cholestatic liver disease. Elafibranor, through activation
of PPAR α,δ targets multiple cell types and biological processes
involved in the pathophysiology of PBC, including cholestasis
(impairment of bile flow in the liver), bile toxicity, inflammation
and fibrosis and bile acid output. In 2019, elafibranor was granted
a Breakthrough Therapy Designation by the U.S Food and Drug
Administration in adults with PBC who have an inadequate response
to ursodeoxycholic acid (UDCA) the existing first-line therapy for
PBC. Elafibranor has not received approval by regulatory
authorities anywhere in the world.
ABOUT
ELATIVE®
ELATIVE is a multi-center, randomized,
double-blind, placebo-controlled Phase III clinical trial, with an
open-label long-term extension (NCT04526665). ELATIVE evaluated the
efficacy and safety of elafibranor 80mg once daily versus placebo
for the treatment of patients with PBC with an inadequate response
or intolerance to UDCA. The trial enrolled 161 patients who were
randomized 2:1 to receive either elafibranor 80mg once daily or
placebo. Patients with an inadequate response to UDCA would
continue to receive UDCA in combination with elafibranor or
placebo, while patients unable to tolerate UDCA would receive only
elafibranor or placebo. Data confirmed the potential for
elafibranor to be an effective new treatment option for PBC, with
13 times more patients achieving a biochemical response, suggesting
an improvement in disease progression, when treated with
elafibranor compared with patients on placebo: 47% placebo-adjusted
difference, elafibranor 80mg (51%) compared with placebo (4%)
(P<0.001).8
Reductions in levels of alkaline phosphatase
(ALP) were rapid, seen as early as Week 4 in the elafibranor group,
and were sustained through Week 52, with a decrease in ALP of 41%
on elafibranor compared with placebo.8 ALP and bilirubin are
important predictors of PBC disease progression. ELATIVE also
investigated the effect of treatment with elafibranor on pruritus
(severe itch), a significant symptom burden amongst people living
with PBC. Findings from the secondary endpoint using the PBC Worst
Itch NRS score, showed a reduction in pruritis for elafibranor,
which was not statistically significant. Data reported from two
separate patient-reported outcome measures demonstrated reductions
in moderate to severe pruritus, which favored elafibranor versus
placebo.8 Elafibranor was well-tolerated in the trial and has a
well-documented safety profile. Adverse events occurring in >10%
of patients and more frequently on elafibranor versus placebo
included abdominal pain, diarrhea, nausea, and vomiting.8
ABOUT GENFIT
GENFIT is a late-stage biopharmaceutical company
dedicated to improving the lives of patients with rare and
life-threatening liver diseases characterized by high unmet medical
needs. GENFIT is a pioneer in liver disease research and
development with a rich history and strong scientific heritage
spanning more than two decades. Today, GENFIT has a growing and
diversified pipeline with programs at various development stages.
The Company’s area of focus is Acute on Chronic Liver Failure
(ACLF). Its ACLF franchise consists of five assets in development:
VS-01, NTZ, SRT-015, CLM-022 and VS-02-HE. These are all based on
differentiated mechanisms of action leveraging complementary
pathways. Other assets target other life-threatening disease
indications such as cholangiocarcinoma (CCA) and Urea Cycle
Disorders (UCD)/Organic Acidemias (OA). GENFIT’s track record in
bringing early-stage assets with high potential to late development
and pre-commercialization stages is highlighted in the successful
52-week Phase 3 ELATIVE® trial evaluating elafibranor in PBC.
Beyond therapeutics, GENFIT’s pipeline also includes a diagnostic
franchise focused on Metabolic dysfunction-associated
steatohepatitis (MASH) previously known as nonalcoholic
steatohepatitis (NASH) and ammonia. GENFIT has facilities in Lille
and Paris (France), Zurich (Switzerland) and Cambridge, MA (USA).
GENFIT is a publicly traded company listed on the Nasdaq Global
Select Market and on compartment B of Euronext’s regulated market
in Paris (Nasdaq and Euronext: GNFT). In 2021, IPSEN became one of
GENFIT’s largest shareholders and holds 8% of the company’s share
capital. For more information, visit www.genfit.com
ABOUT IPSEN
Ipsen is a global biopharmaceutical company with
a focus on bringing transformative medicines to patients in three
therapeutic areas: Oncology, Rare Disease and Neuroscience. Its
pipeline is fueled by external innovation and supported by nearly
100 years of development experience and global hubs in the U.S.,
France and the U.K. Its teams in more than 40 countries and our
partnerships around the world enable us to bring medicines to
patients in more than 100 countries. Ipsen is listed in Paris
(Euronext: IPN) and in the U.S. through a Sponsored Level I
American Depositary Receipt program (ADR: IPSEY). For more
information, visit https://www.ipsen.com/.
GENFIT - FORWARD LOOKING
STATEMENTS
This press release contains certain
forward-looking statements, including those within the meaning of
the Private Securities Litigation Reform Act of 1995 with respect
to GENFIT, including, but not limited to statements about the
potential of elafibranor as a safe and effective second-line
treatment for PBC, the opportunity to manage the disease
progression and the potential of elafibranor to improve pruritus,
reduce cholestatic injury and improve liver function. The use of
certain words, including “believe”, “potential,” “expect”,
“target”, “may” and “will” and similar expressions, is intended to
identify forward-looking statements. Although the Company believes
its expectations are based on the current expectations and
reasonable assumptions of the Company’s management, these
forward-looking statements are subject to numerous known and
unknown risks and uncertainties, which could cause actual results
to differ materially from those expressed in, or implied or
projected by, the forward-looking statements. These risks and
uncertainties include, among other things, the uncertainties
inherent in research and development, including in relation to
safety of drug candidates, cost of, progression of, and results
from, our ongoing and planned clinical trials, review and approvals
by regulatory authorities in the United States, Europe and
worldwide, of our drug and diagnostic candidates, potential
commercial success of elafibranor if approved, exchange rate
fluctuations, our continued ability to raise capital to fund our
development, as well as those risks and uncertainties discussed or
identified in the Company’s public filings with the AMF, including
those listed in Chapter 2 “Main Risks and Uncertainties” of the
Company’s 2022 Universal Registration Document filed with the AMF
on April 18, 2023, which is available on the Company’s website
(www.genfit.com) and on the website of the AMF (www.amf-france.org)
and public filings and reports filed with the U.S. Securities and
Exchange Commission (“SEC”) including the Company’s 2022 Annual
Report on Form 20-F filed with the SEC on April 18, 2023 and
subsequent filings and reports filed with the AMF or SEC, including
the Half-Year Business and Financial Report at June 30, 2023 or
otherwise made public, by the Company. In addition, even if the
Company’s results, performance, financial condition and liquidity,
and the development of the industry in which it operates are
consistent with such forward-looking statements, they may not be
predictive of results or developments in future periods. These
forward-looking statements speak only as of the date of publication
of this document. Other than as required by applicable law, the
Company does not undertake any obligation to update or revise any
forward-looking information or statements, whether as a result of
new information, future events or otherwise.
IPSEN – FORWARD LOOKING
STATEMENTS
The forward-looking statements, objectives and
targets contained herein are based on Ipsen’s management strategy,
current views and assumptions. Such statements involve known and
unknown risks and uncertainties that may cause actual results,
performance or events to differ materially from those anticipated
herein. All of the above risks could affect Ipsen’s future ability
to achieve its financial targets, which were set assuming
reasonable macroeconomic conditions based on the information
available today. Use of the words ‘believes’, ‘anticipates’ and
‘expects’ and similar expressions are intended to identify
forward-looking statements, including Ipsen’s expectations
regarding future events, including regulatory filings and
determinations. Moreover, the targets described in this document
were prepared without taking into account external-growth
assumptions and potential future acquisitions, which may alter
these parameters. These objectives are based on data and
assumptions regarded as reasonable by Ipsen. These targets depend
on conditions or facts likely to happen in the future, and not
exclusively on historical data. Actual results may depart
significantly from these targets given the occurrence of certain
risks and uncertainties, notably the fact that a promising medicine
in early development phase or clinical trial may end up never being
launched on the market or reaching its commercial targets, notably
for regulatory or competition reasons. Ipsen must face or might
face competition from generic medicine that might translate into a
loss of market share. Furthermore, the research and development
process involves several stages each of which involves the
substantial risk that Ipsen may fail to achieve its objectives and
be forced to abandon its efforts with regards to a medicine in
which it has invested significant sums. Therefore, Ipsen cannot be
certain that favorable results obtained during preclinical trials
will be confirmed subsequently during clinical trials, or that the
results of clinical trials will be sufficient to demonstrate the
safe and effective nature of the medicine concerned. There can be
no guarantees a medicine will receive the necessary regulatory
approvals or that the medicine will prove to be commercially
successful. If underlying assumptions prove inaccurate or risks or
uncertainties materialize, actual results may differ materially
from those set forth in the forward-looking statements. Other risks
and uncertainties include but are not limited to, general industry
conditions and competition; general economic factors, including
interest rate and currency exchange rate fluctuations; the impact
of pharmaceutical industry regulation and healthcare legislation;
global trends toward healthcare cost containment; technological
advances, new medicine and patents attained by competitors;
challenges inherent in new-medicine development, including
obtaining regulatory approval; Ipsen’s ability to accurately
predict future market conditions; manufacturing difficulties or
delays; financial instability of international economies and
sovereign risk; dependence on the effectiveness of Ipsen’s patents
and other protections for innovative medicines; and the exposure to
litigation, including patent litigation, and/or regulatory actions.
Ipsen also depends on third parties to develop and market some of
its medicines which could potentially generate substantial
royalties; these partners could behave in such ways which could
cause damage to Ipsen’s activities and financial results. Ipsen
cannot be certain that its partners will fulfil their obligations.
It might be unable to obtain any benefit from those agreements. A
default by any of Ipsen’s partners could generate lower revenues
than expected. Such situations could have a negative impact on
Ipsen’s business, financial position or performance. Ipsen
expressly disclaims any obligation or undertaking to update or
revise any forward-looking statements, targets or estimates
contained in this press release to reflect any change in events,
conditions, assumptions or circumstances on which any such
statements are based, unless so required by applicable law. Ipsen’s
business is subject to the risk factors outlined in its
registration documents filed with the French Autorité des Marchés
Financiers. The risks and uncertainties set out are not exhaustive
and the reader is advised to refer to Ipsen’s latest Universal
Registration Document, available on ipsen.com.
GENFIT CONTACTS
INVESTORS
Jean-Christophe Marcoux – Chief Corporate
Affairs Officer | Tel: +33 3 2016 4000 |
jean-christophe.marcoux@genfit.com
MEDIA
Stephanie Boyer – Press relations | Tel: +333
2016 4000 | stephanie.boyer@genfit.com
IPSEN CONTACTS
INVESTORS
Craig Marks | Tel: +44 (0)7584 349 193 |
craig.marks@ipsen.com
Nicolas Bogler | Tel: +33 6 52 19 98 92 |
nicolas.bogler@ipsen.com
MEDIA
Amy Wolf | Tel: +41 79 576 07 23 |
amy.wolf@ipsen.com
Ioana Piscociu | Tel: +33 6 69 09 12 96 |
Ioana.piscociu@ipsen.com
Anna Gibbins | Tel: +44 (0)7717 801 900 |
anna.gibbins@ipsen.com
GENFIT | 885 Avenue Eugène Avinée, 59120 Loos -
FRANCE | +333 2016 4000 |
www.genfit.com
1 Younossi ZM, et al. 2019. Diagnosis and Management of Primary
Biliary Cholangitis. Am J Gastroenterol. 114(1):48–63.2 European
Association for the Study of the Liver. 2017. EASL Clinical
Practice Guidelines: The diagnosis and management of patients with
primary biliary cholangitis. J Hepatol. 67(1):145-172.3 Galoosian
A, et al. 2020. Clinical updates in primary biliary cholangitis:
trends, epidemiology, diagnostics, and new therapeutic approaches.
J Clin Transl Hepatol. 8(1), pp. 49-60.4 Kumagi T & Heathcote
EJ. 2008. Primary biliary cirrhosis. Orphanet J Rare Di. 3:1.5 Ali
AH, Byrne TJ, Lindor KD. 2015. Orphan drugs in development for
primary biliary cirrhosis: challenges and progress. Orphan Drugs:
Research and Reviews. 5(1), pp..83-97 numbers.6 Corpechot C, et al.
2011. Early primary biliary cirrhosis: biochemical response to
treatment and prediction of long-term outcome. J Hepatol.
55:1361-7.7 Aguilar MT and Chascsa DM. 2020. Update on emerging
treatment options for primary biliary cholangitis. Hepat Med.
Pp.69-77.8 Kowdley. K.V, et al. NEJM. 2023. DOI:
10.1056/NEJMoa2306185
- Ipsen confirms U.S. FDA grants priority review for New Drug
Application for elafibranor for the treatment of rare cholestatic
liver disease, PBC
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