Niche Markets and Rare Diseases

August 2012

Niche Markets and Rare Diseases: Hepatocellular Carcinoma

Report Authors
Karen Pomeranz, M.Sc., Ph.D.
Anouchka Seesaghur, M.B.B.S., M.P.H.
Benjamin Guikema, Ph.D.
  • Pages:142
  • Tables:57
  • Figures:5
  • Citations:122
  • Drugs:31
  • Interviews:8


Hepatocellular carcinoma (HCC) is the most common form of liver cancer and usually develops secondary to liver cirrhosis, which poses a significant medical challenge. Systemic therapy is used for treatment of advanced disease, and this setting is dominated by the small-molecule multitargeted agent sorafenib (Bayer HealthCare and Onyx Pharmaceuticals’ Nexavar), which gained regulatory approval in 2007 in the United and Europe. Sorafenib remains the only targeted therapy approved for treatment of HCC, underscoring the complexity of developing drug therapies for treatment of this disease. Furthermore, HCC is the third most common cause of cancer death globally; hence, a high unmet need for more-efficacious and well-tolerated therapies for treatment of this disease remains.

Questions Answered in This Report:

  *   Emerging therapies are in development for treatment of advanced HCC following disease progression on sorafenib.  Which of these agents are most likely to launch for treatment of this patient population? Which patients will they most likely benefit?

  *   Interviewed experts state that there is a need for additional therapies for treatment of advanced HCC in the first-line setting. Are there emerging drugs that could be used in this patient segment?  Will any drugs challenge sorafenib for patient share?

  *   Drug development efforts are also focusing on early stages of the disease. What classes of agents are most likely to launch in this setting? What proportion of patients will be eligible for treatment with these emerging therapies?


Markets covered: United States, France, Germany, Italy, Spain, United Kingdom.

Primary research: 8 country-specific interviews with expert oncologists/hepatologists.

Epidemiology: Diagnosed incidence segmented by localized, locally advanced, and advanced disease.

Population segments: Localized, locally advanced, advanced first-line, and advanced second-line.

Emerging therapies: Phase II: 13 drugs; Phase III: 9 drugs.

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Mentioned in this report:

  • - 4SC
  • - Abbott
  • - Amgen
  • - ArQule
  • - AstraZeneca
  • - Boehringer Ingelheim
  • - Bristol-Myers Squibb
  • - Celsion
  • - Chugai
  • - Daiichi Sankyo
  • - Eli Lilly
  • - Exelixis
  • - Genentech
  • - Human Genome Sciences
  • - Jennerex Biotherapeutics
  • - Kyowa Hakko Kirin
  • - Merck
  • - MolMed
  • - Novartis
  • - OSI Pharmaceuticals
  • - Pfizer
  • - Polaris Group
  • - Roche
  • - Sanofi
  • - Takeda
  • - Transgene
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