Niche Markets and Rare Diseases

December 2012

Niche Markets and Rare Diseases: Acute Myeloid Leukemia

Report Authors
Khurram Nawaz, M.Sc.
Karen Pomeranz, M.Sc., Ph.D.
Michael Hughes, M.Sc., Ph.D.
Benjamin Guikema, Ph.D.
  • Pages:153
  • Tables:94
  • Figures:4
  • Citations:224
  • Drugs:37
  • Interviews:8


Acute myeloid leukemia (AML) is a biologically heterogeneous group of malignancies of the bone marrow that predominantly affect adult patients aged 60 or older. Although recent advances in the molecular understanding of AML have led to refinements in the classification and prognostication of patients, treatment options have remained virtually unchanged, and all AML subtypes (except acute promyelocytic leukemia [APL]) are treated with the same chemotherapeutic regimens. Current treatment of elderly AML patients is particularly challenging because a substantial proportion of these patients are not eligible for standard intensive chemotherapy or hematopoietic stem-cell transplantation—highlighting the crucial need for efficacious therapies that are less toxic than current myeloablative chemotherapy regimens.

Questions Answered in This Report:

  *   Several emerging therapies, including targeted agents, are in clinical development for elderly patients with AML. Which of these agents are most likely to launch? What are interviewed experts’ opinions of emerging therapies? How rapidly will physicians add these new treatments to their armamentarium?

  *   The hypomethylating agent Dacogen (Eisai/Johnson & Johnson/Astex Pharmaceuticals’ decitabine) earned European approval in September 2012 for the treatment of elderly patients with AML who are not eligible for standard induction chemotherapy. What is the role of hypomethylating agents in the current treatment of AML? What are thought leaders’ perceptions of Dacogen? Does European approval of Dacogen affect physicians’ perceptions of the hypomethylating agent Vidaza (Celgene’s azacitidine)?

  *   Interviewed experts state that there is a great need for efficacious therapies to treat relapsed/refractory (R/R) AML. Can any of the emerging drugs be used in this patient population? What are interviewed experts’ opinions of emerging therapies for R/R AML? What challenges and opportunities remain?


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

Primary research: Eight country-specific interviews with expert hematological oncologists.

Epidemiology: Diagnosed incidence of AML, diagnosed incidence of APL.

Population segments: First-line AML, relapsed/refractory AML, first-line APL, relapsed/refractory APL.

Emerging therapies: Phase II: 13 drugs; Phase III: 5 drugs.

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Mentioned in this report:

  • - 4SC
  • - Akinion Pharmaceuticals
  • - Ambit Biosciences
  • - Astellas Pharma
  • - Astex Pharmaceuticals
  • - Baxter
  • - Bayer
  • - BioSante Pharmaceuticals
  • - Boehringer Ingelheim
  • - Bristol-Myers Squibb
  • - Celator Pharmaceuticals
  • - Celgene
  • - Cell Therapeutics
  • - Cephalon
  • - Chroma Therapeutics
  • - Clavis Pharma
  • - Cyclacel Pharmaceuticals
  • - Daiichi Sankyo
  • - Dava Pharmaceuticals
  • - Dendreon
  • - Eisai
  • - Formula Pharmaceuticals
  • - Genzyme
  • - GlaxoSmithKline
  • - Janssen
  • - Janssen-Cilag
  • - Johnson & Johnson
  • - Meda
  • - Merck & Co.
  • - Merck KGaA
  • - Novartis
  • - Pfizer
  • - Proacta
  • - ProQinase GmbH
  • - Roche
  • - Sanofi
  • - Stemline Therapeutics
  • - Sunesis Pharmaceuticals
  • - Takeda
  • - TetraLogic Pharmaceuticals
  • - Teva
  • - The Vaccine Company
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