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Non-alcoholic steatohepatitis (NASH) Market and Forecast Analysis to 2038

  • Report
  • 102 Pages
  • January 2021
  • Region: Global
  • Citeline
  • ID: 4775300
Disease Overview

Non-alcoholic fatty liver disease (NAFLD) is a hepatic condition closely associated with metabolic syndrome, and non-alcoholic steatohepatitis (NASH) is a segment of NAFLD. Both NAFLD and NASH patients display hepatic steatosis, and do not drink more than recommended guideline amounts of alcohol. Hepatocellular injury may or may not be present in NAFLD patients; however, NASH is characterized by the presence of hepatocellular injury marked by hepatic steatosis, inflammation, and/or fibrosis.

Latest Key Takeaways

  • 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.

Table of Contents

  • Latest key takeaways

  • Definition
  • Symptoms
  • Diagnosis
  • Patient segmentation

  • Prevalence methodology

  • 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

  • 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

  • Sponsors by status
  • Sponsors by phase
  • Recent events

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

  • 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

  • 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)

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

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

Figure 1: Trends in prevalent cases of NASH, 2018–27
Figure 2: Overview of pipeline drugs for NASH in the US
Figure 3: Pipeline drugs for NASH, by company
Figure 4: Pipeline drugs for NASH, by drug type
Figure 5: Pipeline drugs for NASH, by classification
Figure 6: Probability of success in the endocrine pipeline
Figure 7: Clinical trials in NAFLD
Figure 8: Top 10 drugs for clinical trials in NAFLD
Figure 9: Top 10 companies for clinical trials in NAFLD
Figure 10: Trial locations in NAFLD
Figure 11: NAFLD trials status
Figure 12: NAFLD trials sponsors, by phase
Figure 13: The publisher’s drug assessment summary for NASH
Figure 14: Current dynamics in NASH
Figure 15: Market opportunity in NASH
Figure 16: Threat of substitution in NASH
Figure 17: Payer pressure/buyer power in NASH
Figure 18: Future trends in NASH (1 of 2)
Figure 19: Future trends in NASH (2 of 2)
Figure 20: LPCN 1144 for NASH (January 12, 2021): Phase II - LiFT (Biopsy-Confirmed NASH)
Figure 21: Icosabutate for NASH (January 8, 2021): Phase II - ICONA
Figure 22: PXL770 for NASH (October 1, 2020): Phase IIa - POC
Figure 23: BIO89-100 for NASH (September 14, 2020): Phase Ib/IIa - MAD
Figure 24: Aldafermin for NASH (August 29, 2020): Phase II - 15-0105
Figure 25: Nidufexor for NASH (August 28, 2020): Phase II - Safety/Efficacy
Figure 26: VK2809 for NASH (August 28, 2020): Phase II - Hyperlipidemia/NAFLD
Figure 27: Elobixibat for NASH (August 18, 2020): Phase II - NAFLD/NASH
Figure 28: Efruxifermin for NASH (June 30, 2020): Phase IIa - BALANCED
Figure 29: TVB-2640 for NASH (June 17, 2020): Phase II - FASCINATE-1
Figure 30: Cotadutide for NASH (June 15, 2020): Phase II - Hepatic Glycogen Metabolism Effect
Figure 31: Lanifibranor for NASH (June 15, 2020): Phase IIb - NATIVE
Figure 32: Elafibranor for NASH (May 11, 2020): Phase III - RESOLVE-IT
Figure 33: Ozempic for NASH (May 6, 2020): Phase II - NN9931-4296
Figure 34: Key upcoming events in NASH
Table 1: Fibrosis staging in NAFLD using the Brunt et al. system adapted by the NASH CRN Pathology Committee
Table 2: Guideline recommendations on lifestyle interventions
Table 3: Summary of pharmacological recommendations for NASH/NAFLD from US, EU, and Japanese guidelines
Table 4: Prevalent cases of NASH, 2018–27
Table 5: Pipeline drugs for NASH in the US
Table 6: Forecasted global sales, by drug ($m), 2021–25
Table 7: LPCN 1144 for NASH (January 12, 2021)
Table 8: Icosabutate for NASH (January 8, 2021)
Table 9: Multiple Drugs for NASH (November 15, 2020)
Table 10: PXL770 for NASH (October 1, 2020)
Table 11: BIO89-100 for NASH (September 14, 2020)
Table 12: Aldafermin for NASH (August 29, 2020)
Table 13: Nidufexor for NASH (August 28, 2020)
Table 14: VK2809 for NASH (August 28, 2020)
Table 15: Elobixibat for NASH (August 18, 2020)
Table 16: Efruxifermin for NASH (June 30, 2020)
Table 17: Namodenoson for NASH (June 30, 2020)
Table 18: Ocaliva for NASH (June 29, 2020)
Table 19: TVB-2640 for NASH (June 17, 2020)
Table 20: Cotadutide for NASH (June 15, 2020)
Table 21: Lanifibranor for NASH (June 15, 2020)
Table 22: Elafibranor for NASH (May 11, 2020)
Table 23: Seladelpar for NASH (May 8, 2020)
Table 24: Ozempic for NASH (May 6, 2020)