Unmet Needs
Unmet Needs 2016: Non-Alcoholic SteatoHepatitis (NASH)
October 20, 2016

Rising Stars Outlook 2016: New Emergence of Potential Tx & Dx Options could transform the Current Landscape for Liver Diseases (NASH) – Research and Markets


Non-Alcoholic SteatoHepatitis (NASH) is an unmet medical need and is becoming the leading cause of liver transplant. No FDA-approved therapies and a meager diagnosis, it poses a unique challenge in the design of studies for NASH. Therefore, there is a worldwide need for new therapies to treat the global epidemic of NASH and the cost to society will be huge if it is not addressed in the upcoming years. Besides, scarcity of treatment options, there is also an urgent unmet need to develop biomarkers that facilitate the diagnosis, identification of populations at risk, assessment of disease progression or regression, and/or response to treatment. The potential market is gigantic with analysts predicting north of $35bn by 2025.

Many small players are currently developing drugs on their own and with such a large unmet need, market potential and disease heterogeneity, we expect many Giants to join this race and thus, we can see more Licensing deals/ M&A activity on the horizon.

NAFLD/NASH – Race to the Market: Strategies and Tactics to Address Unmet Needs


Although the NAFLD/NASH race to the market is well underway, a number of challenges face drug development programs; biopsy requirements, lack of validated surrogate biomarkers, and no clear regulatory guidelines. Overcoming these obstacles will not only benefit the pharmaceutical and biotech industry to bring new therapies to market, but will ensure that the millions of NAFLD/NASH patients are treated or cured of this chronic liver disease.

Corporate Presentation – Galectin Therapeutics


“NASH is a silent public health time bomb that may soon overload our health care system with hospital admissions. By 2020, it is expected to eclipse hepatitis C as the leading reason for liver transplants.”

NASH- Genfit


NASH: A glance at the landscape of pharmacological treatment


Tobira Therapeutics: Corporate Presentation


Obeticholic Acid for the Treatment of Nonalcoholic Steatohepatitis: Comparative Clinical Effectiveness and Value Evidence Report


Emerging Anti-fibrotic Therapies in the Treatment of Non-alcoholic Steatohepatitis. M. Noureddin; Q. M. Anstee; R. Loomba. Aliment Pharmacol Ther. 2016;43(11):1109-1123.


Screening Strategies for Nonalcoholic Steatohepatitis in High-Risk Individuals: Trimming Away the Fat.


The Development Landscape for Non-Alcoholic Steatohepatitis


Q&A with Claudia Filozof, MD, PhD, Senior Medical Director in the Phase II-IV Cardiovascular/Metabolic Group and Richard Williams, PhD, JD, Executive Strategist, Global Regulatory Affairs

What do you see as the most critical challenges in NASH development?

Claudia: Companies still need to use an invasive method for the primary and many secondary endpoints, but patients may not be willing to have multiple liver biopsies. Also, completing a clinical outcomes study requires at least five or six years. Another important challenge is the natural history of the disease: many patients spontaneously regress, which makes the placebo behavior ,in some cases, difficult to predict.

Richard: One risk involves reimbursement. Companies have to consider whether health authorities in other countries would be willing to pay for this drug and what evidence they would want to see. The best strategy is to have dialogues with health technology assessment groups early in the process, then incorporate clinical endpoints to demonstrate that the treatment has a real benefit for patients in the eyes of the payers.