Global Non-alcoholic Steatohepatitis (NASH) Market 2021-2038: Epidemiology, Regulatory Events, Deals, Clinical Trial Landscape, Drug Assessment Model, Future Trends, Unmet Needs

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Mar 24, 2021, 09:30 ET

DUBLIN, March 24, 2021 /PRNewswire/ -- The "Non-alcoholic steatohepatitis (NASH) Market and Forecast Analysis to 2038" report has been added to ResearchAndMarkets.com's offering.

There are currently no disease-specific approved therapies for non-alcoholic steatohepatitis (NASH), which creates a significant unmet need due to the high clinical and economic burden on healthcare systems and the large prevalent population. NASH is expected to become the leading indication for liver transplant in the US in the next few years, which increases the urgency of finding an efficacious therapy.

The slow and largely unsuccessful path to achieving a promising treatment has been hindered by the complicated and poorly understood pathophysiology of NASH. Due to the multifactorial nature of the disease, there is a diverse range of modes of action in the pipeline and substantial interest in combination therapies.

The publisher estimates that in 2018, there were approximately 869.3 million prevalent cases of NASH worldwide in adults aged ?20 years, and forecasts that number to increase to 965.4 million prevalent cases by 2027.

In order to gain accelerated approval by the US Food and Drug Administration (FDA), one of the following endpoints must be met: 1) improvement of 1 stage in fibrosis with no worsening of NASH; 2) improvement in NASH resolution with no worsening of fibrosis.

While the FDA only requires achievement of one of the above endpoints, the European Medicines Agency (EMA) requires efficacy on both of these endpoints in a co-primary fashion, which could restrict or delay approvals in the five major European markets (France, Germany, Italy, Spain, and the UK). Notably, the EMA's guidance is still in draft form, therefore could be changed in response to industry lobbying before finalization.

Given the lack of any marketed NASH therapies to date, the NASH market landscape is expected to grow dramatically over the forecast period, driven by a surge of new product approvals in the space.

Despite Ocaliva's recent rejection by the FDA, the publisher expects that the drug will be approved in Q2 2022 following Intercept's anticipated NDA resubmission with additional data requested by the FDA. However, this still means the drug will lose its opportunity to monopolize the market for a significant period of time.

While the FDA has described an imbalance in Ocaliva's risk/benefit profile as the reason for the drug's rejection, increasing skepticism over the reliability of surrogate endpoints following large trial failures could have influenced its decision. It is also possible that the FDA is retrospectively raising the bar for accelerated approval (having previously agreed on the required surrogate endpoints for accelerated approval), and as Ocaliva was the first NDA submitted in NASH, the FDA may be acting particularly cautiously to set a precedent for future NASH therapies.

Although first approvals in the market will experience rapid uptake, label recommendations will initially restrict use to F2/F3 NASH patients, reflecting the enrollment criteria of Phase III trials.

High prices of new therapies will translate into high revenues; however, strict reimbursement criteria will limit market access for prohibitively priced therapies, including Ocaliva, which is expected to have a premium price while it monopolizes the market.

The anticipated requirement for biopsy-confirmed NASH is expected to be the major barrier to treatment access. Therefore, in the short term, treatments will predominantly be prescribed by specialists such as gastroenterologists and hepatologists, which will limit the ease of accessibility of treatment.

Clinical trials focusing on F4 patients with compensated cirrhosis remain an area of unmet need. Companies are reluctant to pursue programs in this space as cirrhosis has predominantly been viewed as irreversible; however, given that these patients are closely associated with negative hepatic outcomes, they are considered the highest priority to treat.

Another challenge that will remain once the first therapy is approved for NASH is how to measure a treatment benefit in the commercial setting, as using biopsies will not be feasible on such a large scale; however, there are no reliable non-invasive replacements yet available.

Numerous high-impact upcoming events for drugs in the NASH space are expected during 2021, including a CHMP opinion on Ocaliva.

Key Topics Covered:

OVERVIEW

Latest key takeaways

DISEASE BACKGROUND

Definition
Symptoms
Diagnosis
Patient segmentation

TREATMENT

EPIDEMIOLOGY

Prevalence methodology

PIPELINE DRUGS

KEY REGULATORY EVENTS

Intercept's NASH Plans For Better Alignment With FDA
Intercept Unlikely To Refile OCA For NASH Before Late 2021
BTD Confirms Lanifibranor As Leader Of The NASH Pack (For Now)
Intercept's CRL Just Continues Upheaval In NASH
Intercept Eyes EU NASH Market
Could Postponement of Intercept's Expected NASH Panel Impact Its PDUFA Date?
Genfit Delays NASH Data While It Confers With US FDA On Methodology

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

Deal Watch: Merck & Co. Looks To Advance NASH Ambitions With Aligos
Israel's Galmed, MyBiotics Team To Improve NASH Therapeutic Response
Novartis Licenses NASH Candidate To Boston Pharmaceuticals
LG Chem Expands NASH Pipeline Via License Deal With TransThera
Takeda Seeks Direct Anti-Fibrotic Targets From Engitix
Merck Picks Up Hanmi's Dual Agonist Efinopegdutide, But For NASH
Promethera Links With Hao Tian On JV For NASH, ACLF, Hepatic Cancer

CLINICAL TRIAL LANDSCAPE

Sponsors by status
Sponsors by phase
Recent events

DRUG ASSESSMENT MODEL

Farnesoid X receptor (FXR) agonists
FXR agonist and acetyl-CoA carboxylase (ACC) allosteric inhibitor
Chemokine receptor CCR2/5 antagonist
Thyroid-hormone selective agonists
Fibroblast growth factor (FGF) analogs
Glucagon-like peptide-1 (GLP-1) agonist
Peroxisome proliferator-activated receptor (PPAR) agonists

MARKET DYNAMICS

FUTURE TRENDS

Payer restrictions will limit market access for prohibitively priced first-generation NASH agents
Ocaliva's advantage of monopolizing the market has been eliminated following CRL
Increased competition from new product launches will shape the NASH market
GLP-1 agonists will experience rapid uptake in diabetic NASH patients
No therapy for F4 NASH patients on the horizon
Use of combination therapies will be restricted to severe patients due to prohibitive pricing

CONSENSUS FORECASTS

RECENT EVENTS AND ANALYST OPINION

LPCN 1144 for NASH (January 12, 2021)
Icosabutate for NASH (January 8, 2021)
Multiple Drugs for NASH (November 15, 2020)
PXL770 for NASH (October 1, 2020)
BIO89-100 for NASH (September 14, 2020)
Aldafermin for NASH (August 29, 2020)
Nidufexor for NASH (August 28, 2020)
VK2809 for NASH (August 28, 2020)
Elobixibat for NASH (August 18, 2020)
Efruxifermin for NASH (June 30, 2020)
Namodenoson for NASH (June 30, 2020)
Ocaliva for NASH (June 29, 2020)
TVB-2640 for NASH (June 17, 2020)
Cotadutide for NASH (June 15, 2020)
Lanifibranor for NASH (June 15, 2020)
Elafibranor for NASH (May 11, 2020)
Seladelpar for NASH (May 8, 2020)
Ozempic for NASH (May 6, 2020)

KEY UPCOMING EVENTS

KEY OPINION LEADER INSIGHTS

NASH market
Ocaliva
Resmetirom
FGF analogs
Combination therapies
Endpoints
Prescribing trends
F4 patients
Pricing
Biopsies and non-invasive tools

UNMET NEEDS

Pharmacological treatment
Clinical trials in advanced NASH patients
Non-invasive diagnostic tools

BIBLIOGRAPHY

APPENDIX

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