The European Commission has granted conditional approval
for Ipsen's IQIRVO (elafibranor) for the treatment of primary
biliary cholangitis in adults, marking the first new therapy for
this condition to be approved in the EU in almost ten years. With
this approval, other companies such as CymaBay Therapeutics, Zydus
Therapeutics, GlaxoSmithKline, and others also geared up to bring
their candidates into the primary biliary cholangitis
market.
LAS VEGAS, Oct. 2, 2024 /PRNewswire/ -- Primary biliary
cholangitis (PBC), previously referred to as primary biliary
cirrhosis, is a long-term liver condition characterized by the
gradual destruction of the small bile ducts within the liver, known
as intrahepatic bile ducts. Over time, prolonged bile flow
obstruction can result in cirrhosis and the development of portal
hypertension.
As per DelveInsight analysis, in 2023, the total diagnosed
prevalent cases of primary biliary cholangitis in the 7MM were
approximately 298K. These
cases are projected to increase further during the forecast period
(2024–2034). In 2023, the United
States had the highest proportion of diagnosed prevalent
cases of primary biliary cholangitis among the 7MM, comprising
approximately 48% of the total cases.
DelveInsight's epidemiology model for primary biliary
cholangitis estimates a higher prevalence among females across the
7MM. Specifically, in the US in 2023, it suggests approximately
25K cases among males and
117K cases among females,
indicating a female predominance.
The primary treatment objectives are to reduce the progression
rate of the disease and manage symptoms such as itching,
osteoporosis, and dry eye/mouth (sicca syndrome). Liver
transplantation remains the only life-saving option.
Ursodeoxycholic acid (UDCA) is the main medication used to
slow disease progression.
Patients in the early stages often experience clinical,
biochemical, and histological improvements. Studies suggest UDCA
postpones the need for liver transplantation and extends survival,
though its effectiveness in advanced stages like cirrhosis is
uncertain. While initially approved by the US FDA for treating
primary biliary cholangitis, UDCA now has generic versions
available.
The use of corticosteroids in patients without signs of
autoimmune hepatitis remains a subject of ongoing research. Studies
have shown that combining UDCA with dexamethasone can enhance the
activity of the anion exchanger from two alternate promoters in
liver cells in vitro. Budesonide is being considered due to its
extensive first-pass hepatic metabolism, which may help minimize
side effects. Corticosteroids may help relieve symptoms and improve
biochemical and histological outcomes, though
corticosteroid-induced osteoporosis is a major concern.
The criteria for liver transplantation in patients with primary
biliary cholangitis are generally the same as those for other
chronic liver diseases. However, severe and unmanageable itching is
a unique reason for considering a transplant. Fatigue, which may
continue after transplantation, is not regarded as an indication
for the procedure. Liver transplantation for PBC patients typically
offers a better prognosis than for most other chronic liver
diseases, with a 5-year survival rate of 84–87% and a 10-year
survival rate of 79–84%.
In May 2016, the US FDA granted
accelerated approval for OCALIVA (obeticholic acid) as a
treatment for primary biliary cholangitis. It is approved for use
alongside UDCA in adults who do not respond adequately to UDCA, or
as a standalone treatment for those who cannot tolerate
UDCA.
OCALIVA is a semi-synthetic bile acid analog with the chemical
structure 6α-ethyl-chenodeoxycholic acid. OCALIVA remains the only
approved second-line therapy for this condition. Later, in
December 2016, the European
Commission gave conditional approval for OCALIVA for the same
uses—either in combination with UDCA or as monotherapy for adults
unable to tolerate UDCA.
Learn more about the FDA-approved primary biliary cholangitis
drugs @ Drugs for Primary Biliary Cholangitis Treatment
Recently, the European Commission has granted conditional
approval for IQIRVO (elafibranor) 80mg tablets for treating
primary biliary cholangitis. It can be used alongside
ursodeoxycholic acid in adults who have an inadequate response to
UDCA or as a standalone therapy for those who cannot tolerate UDCA.
This decision follows a positive recommendation from the Committee
for Medicinal Products for Human Use (CHMP) of the European
Medicines Agency (EMA) on July 26,
2024, and the US FDA's Accelerated Approval granted on
June 10, 2024.
Elafibranor, a pioneering molecule, has been marketed and
commercialized in the US by Ipsen under the brand name
IQIRVO since June 2024. It was
originally developed by GENFIT, from its discovery through the
completion of a 52-week Phase III clinical trial. In 2021, Ipsen
obtained exclusive global rights to elafibranor, except for
China, Hong Kong, Taiwan, and Macau, through a licensing agreement with
GENFIT.
The EU approval was based on favorable outcomes from the
Phase III ELATIVE trial, which demonstrated a 47% clinical
benefit of IQIRVO combined with UDCA compared to placebo
plus UDCA on the composite primary endpoint. The main objective for
cholestasis response was defined as alkaline phosphatase (ALP)
being less than 1.67 times the upper limit of normal (ULN), with a
≥15% reduction in ALP and total bilirubin levels within the normal
range after one year. Additionally, the treatment showed a
reduction in pruritus when compared to placebo.
Additionally, in August 2024,
Gilead Sciences, Inc. announced that the FDA has granted
accelerated approval for LIVDELZI (seladelpar) to treat primary
biliary cholangitis. This treatment can be used alongside
ursodeoxycholic acid in adults who have not responded adequately to
UDCA or as a standalone therapy for those unable to tolerate UDCA.
Livdelzi is not advised for patients with or who develop
decompensated cirrhosis.
Seladelpar (MBX-8025) is a potent and selective
PPARδ agonist, administered orally, and is currently being
developed for patients with PBC who either do not respond to or
cannot tolerate UDCA. In March 2024,
Gilead Sciences completed its acquisition of CymaBay and announced
the validation of a Marketing Authorization Application (MAA) for
Seladelpar by the European Medicines Agency (EMA).
Seladelpar has been granted Breakthrough Therapy Designation
(BTD) by the US FDA, PRIME status by the EMA, and
Orphan Drug Designation (ODD) from both agencies, which may
accelerate the approval process.
To know more about primary biliary cholangitis treatment
options, visit @ New Treatment for Primary Biliary
Cholangitis
With this approval, several companies have also geared up to
bring their lead assets into the market. The expected launch of
emerging therapies, such as Saroglitazar Magnesium (Zydus
Therapeutics), Linerixibat (GlaxoSmithKline),
Setanaxib (Calliditas Therapeutics AB), Bezafibrate +
obeticholic acid (Intercept Pharmaceuticals/Alfasigma),
Volixibat (Mirum Pharmaceuticals), EP547 (Escient
Pharmaceuticals), and others are expected to create a positive
impact on the market. Other therapies in the early stages of the
trial are also being developed.
Discover which therapies are expected to grab major primary
biliary cholangitis market share @ Primary Biliary Cholangitis
Market Report
Saroglitazar (LIPAGLYN) comprises two primary types of
PPAR agonists: PPARα (alpha) and PPARγ (gamma). This medication
simultaneously reduces lipid and glucose levels, effectively
decreasing elevated triglycerides and blood sugar while enhancing
insulin sensitivity. The US FDA has awarded Saroglitazar orphan
drug designation (ODD) and fast track designation (FTD)
for primary biliary cholangitis (PBC). It is presently undergoing
Phase II/III trials for this condition.
Linerixibat (GSK2330672) is a medication under
development for patients with primary biliary cholangitis who
suffer from itching. It is a small molecule inhibitor of the ileal
bile acid transporter (IBAT) that is minimally absorbed and taken
as an oral tablet. By preventing the reabsorption of bile acids in
the small intestine, linerixibat lowers the levels of pruritic bile
acids in the bloodstream. In 2019, the FDA designated it as an
orphan drug (ODD). It is currently being evaluated in a
Phase III trial for treating cholestatic pruritus in individuals
with PBC.
Discover more about drugs for primary biliary
cholangitis in development @ Primary Biliary Cholangitis
Clinical Trials
The anticipated launch of these emerging therapies for primary
biliary cholangitis are poised to transform the market
landscape in the coming years. As these cutting-edge therapies
continue to mature and gain regulatory approval, they are expected
to reshape the primary biliary cholangitis market landscape,
offering new standards of care and unlocking opportunities for
medical innovation and economic growth.
DelveInsight estimates that the primary biliary cholangitis
market saw a significant emergence of a dominant treatment
modality, valued at USD 1.1
billion in the 7MM. This modality likely reflects
advancements in therapeutic approaches such as first-line agents
like UDCA, second-line treatments like obeticholic acid, and
potentially emerging biologics or combination therapies aiming to
address the complex pathophysiology of PBC, thus enhancing patient
outcomes and market share.
DelveInsight's latest published market report titled as
Primary Biliary Cholangitis Market Insight, Epidemiology, and
Market Forecast – 2034 will help you to discover which
market leader is going to capture the largest market share. The
report provides comprehensive insights into the primary biliary
cholangitis country-specific treatment guidelines, patient pool
analysis, and epidemiology forecast to help understand the key
opportunities and assess the market's underlying potential. The
primary biliary cholangitis market report proffers
epidemiological analysis for the study period 2020–2034 in the 7MM
segmented into:
- Total Diagnosed Prevalent cases of Primary Biliary
Cholangitis
- Gender-specific Diagnosed Prevalent cases of Primary Biliary
Cholangitis
- Age-specific Diagnosed Prevalent cases of Primary Biliary
Cholangitis
The report provides an edge while developing business strategies
by understanding trends shaping and driving the 7MM primary biliary
cholangitis market. Highlights include:
- 11-year Forecast
- 7MM Analysis
- Epidemiology-based Market Forecasting
- Historical and Forecasted Market Analysis up to 2034
- Emerging Drug Market Uptake
- Peak Sales Analysis
- Key Cross Competition Analysis
- Industry Expert's Opinion
- Access and Reimbursement
Download this primary biliary cholangitis market report to
assess the epidemiology forecasts, understand the patient journeys,
know KOLs' opinions about the upcoming treatment paradigms, and
determine the factors contributing to the shift in the primary
biliary cholangitis market. Also, stay abreast of the mitigating
factors to improve your market position in the primary biliary
cholangitis therapeutic space.
Related Reports
Primary Biliary Cholangitis Pipeline
Primary Biliary Cholangitis Pipeline Insight –
2024 report provides comprehensive insights about the
pipeline landscape, pipeline drug profiles, including clinical and
non-clinical stage products, and the key PBC companies,
including Genfit, Zydus Discovery, Ohara Pharmaceutical, Chia
Tai Tianqing Pharmaceutical Group Co., Ltd., Novartis, CymaBay
Therapeutics, among others.
Primary Biliary Cholangitis Epidemiology
Forecast
Primary Biliary Cholangitis Epidemiology Forecast –
2032 report delivers an in-depth understanding of the
disease, historical and forecasted epidemiology, and PBC
epidemiology trends.
Liver Cirrhosis Market
Liver Cirrhosis Market Insights, Epidemiology, and Market
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the disease, historical and forecasted epidemiology, as well as the
market trends, market drivers, market barriers, and key liver
cirrhosis companies, including Madrigal Pharmaceuticals, Inc.,
Galectin Therapeutics Inc., Grifols Therapeutics LLC, CymaBay
Therapeutics, Akero Therapeutics, Inc., NGM Biopharmaceuticals,
Inc., Gilead Sciences, Novo Nordisk A/S, Cellaion, Promethera
Therapeutics, Lipocine Inc., Bristol Myers Squibb, Prism Pharma,
Ohara Pharmaceutical, Shionogi, among others.
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Liver Cirrhosis Pipeline Insight – 2024 report
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pipeline drug profiles, including clinical and non-clinical stage
products, and the key liver cirrhosis companies, including
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Pharmaceuticals, Boehringer Ingelheim, Ohara Pharmaceutical, Ocelot
Bio, Calliditas Therapeutics, Galecto Biotech, Pharmicell,
among others.
About DelveInsight
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