End of Forecast Surge in Sales for NASH from 2012–2017

Nonalcoholic Steatohepatitis (NASH) Market

New research estimates the 2012 off-label sales for nonalcoholic steatohepatitis (NASH) to be approximately $233m across the US and five major European markets of France, Germany, Italy, Spain, and the UK.

By forecast end in 2017, GlobalData expects sales to grow to about $1.36bn, with a compound annual growth rate (CAGR) of 42.2% across the 6MM. We expect the US to claim the most sales, contributing $1.28bn of global sales and a CAGR of 43.8%.

Major drivers of growth in the NASH market are attributed to two factors:

The growing rate of obesity and diabetes globally.
The launch of Genfit’s GFT505 and Intercept/DSP’s obeticholic acid (OCA) in 2017. In OCA’s favor, its Phase II/IIb trial was stopped early in January 2014, after a planned interim analysis showed that the primary endpoint of the study had been met. While for GFT505, Genfit is aligning itself to register GFT505 among the first approved therapies for NASH and the company received Fast Track designation for the GFT505 NASH program on February 14, 2014.
We expect the US’ growth will be due in part to the launch of both of these products, while growth in the 5EU will be predominantly driven by GFT505 during the forecast period.

Major barriers to the growth of the NASH market are attributed to:

Slow development of therapies specific for NASH.
Unclear understanding of the pathophysiology of NASH.
Inefficient diagnostic tools for NASH that may lead to delayed diagnosis of NASH.

Emerging Market Players Employing Strategies to Improve Diagnostics and Trial Designs

The NASH market is virtually vacant aside from off-label therapies that do not provide a “one size fits all” approach. The potential for market leaders to emerge is wide open to all contenders. We expect Gilead and Novo Nordisk to gain market entry after the end of the forecast period in 2017; however, with their market knowledge, their products simtuzumab and liraglutide should do well commercially.

As for newcomers Genfit and Intercept/DSP, fame will be claimed since their products will likely launch at the end of the forecast in Q4 2017, thus giving them an early foothold in the NASH market space.

Since there is no cure or one specific therapy currently available for NASH, R&D strategies in this market space are complicated. Complications range from unraveling the pathophysiology of NASH, to designing diagnostic tools for more accurate diagnosis and staging, to the design of clinical trials. As our expert leaders have agreed, NASH needs an approved therapy that is cost-effective and touts a desirable efficacy and safety profile.

Vast Unmet Needs in the NASH Market:

The level of unmet needs for NASH, including both environmental and clinical, is high and includes patient awareness, physician education, and approved therapies specific to NASH. These issues compound the reasons why the NASH market space is very sparse. A series of unknowns exist for the disease, due to its heterogeneous nature, and its slow progression poses problems for drug development. Vitamin E, the current gold-standard therapy, dominates what little drug therapy NASH market there is. We expect both Genfit’s small molecule GFT505 and Intercept Pharmaceuticals/DSP’s small molecule OCA to claim patient share in all subpopulations of NASH.

These products will likely solve the top unmet need – the dire need for an approved therapy for NASH.

Opportunities Will Remain for Tools that Provide Accurate Diagnosis:

There is a major opportunity for new entrants to design better biomarkers than the current liver enzymes: alanine aminotransferase (ALT) and aspartate aminotransferase (AST) as non-invasive surrogates for Nonalcoholic Fatty Liver Disease (NAFLD). Testing may also include cytokeratin-18 (CK18), which may provide clues to hepatocyte cell death. In addition, markers that can assess liver stages are needed, since CK18 is also elevated in other liver conditions.

Currently, NASH patients may be diagnosed by chance during routine serological examination, or via invasive liver biopsy. Most NASH patients will be in the mid/late stage of the disease once diagnosed. A more accurate diagnostic tool for determining the extent of fibrosis in NASH patients will also be paramount. That said, we expect these opportunities to remain unmet during the five-year forecast period from 2012–2017.

2017 Launches of Novel Pipeline Products Will Forever Change the Future NASH Treatment Landscape:

Twelve molecules of interest across all stages of development make up the pipeline for NASH. However, GlobalData believes there are four therapies with the potential to revolutionize the treatment paradigm for NASH in the near term. These four novel, potential first-in-class therapies, which are antifibrotic and antidiabetic by therapeutic class, will target subpopulations (obese, diabetic, and “other” or healthy donors) within NASH.

The four most promising NASH pipeline products are:

Genfit’s GFT505: anticipated to launch in Q4 2017 across the 6MM.
Intercept Pharmaceuticals / Dainippon Sumitomo Pharma’s obeticholic acid (INT-747/DSP-1747): anticipated to launch in Q4 2017 in the US, with an expected launch in the EU after the forecast period of 2012–2017.
Gilead’s simtuzumab: anticipated to launch after the forecast period of 2012–2017.
Novo Nordisk’s liraglutide (Victoza): the marketed type 2 diabetes brand is anticipated to launch (with NASH as a new indication) after the forecast period of 2012–2017.

These information are basesd on our recent market research report: OpportunityAnalyzer: Nonalcoholic Steatohepatitis (NASH) - Opportunity Analysis and Forecasts to 2017 - Event-Driven