Niche Markets and Rare Diseases

December 2012

Niche Markets and Rare Diseases: Myelodysplastic Syndromes

Report Authors
Benjamin Guikema, Ph.D.
Christopher Craggs, M.Sc.
  • Pages:116
  • Tables:42/28
  • Figures:14
  • Citations:56
  • Drugs:36
  • Interviews:7


Myelodysplastic syndromes (MDS) are heterogeneous in cause and manifestations but share the common features of aberrant hematopoiesis and deteriorating cytopenias. Patients with MDS suffer from anemia, neutropenia, and thrombocytopenia, all of which lead to outcomes such as extreme fatigue, high rates of infections, excessive bleeding, and shortened life span; MDS patients also are at risk of their disease transforming into acute myeloid leukemia.

<>MDS patients are classified by morphology and prognosis into lower-risk or higher-risk MDS categories. Only three drugs have been approved in the United States or Europe specifically for MDS, and second-line therapies do not exist beyond clinical trials or best supportive care (BSC). Allogeneic hematopoietic stem cell transplant (HSCT) is the only potentially curative therapeutic option; however, approximately three-quarters of the MDS patient population are ineligible for HSCT due to older age and poor overall health status. MDS patients need new treatments that are targeted at specific patient subsets, particularly transfusion-dependent patients and the higher-risk patients who are refractory to hypomethylating agents (HMA). Clinical trials for such therapies should focus on improved overall survival in higher-risk patients and improved quality of life (e.g., reduced transfusion dependence or reduced rate of infections) in lower-risk patients.

Questions Answered in This Report:

  *   There is a high level of unmet need for specific subsets of MDS patients. For example, HMA-refractory higher-risk patients have no second-line therapeutic options outside of clinical trials or BSC; lower-risk patients have a longer expected lifespan than higher-risk patients but still suffer from significantly reduced quality of life, requiring frequent transfusions, antibiotics, and hospitalizations. Are there any treatments in clinical development that are likely to address the needs of these patients? Which patient subsets have the highest level of unmet need?

  *   The pathology of MDS is not well understood and the disease is heterogeneous. What are the new avenues of research and understanding of MDS pathophysiology? What new mechanisms of action are in clinical development? Do they offer advantages over current first-line therapies or focus on combination-therapy opportunities? Will any of these emerging MDS therapies launch between 2012 and 2021? In which patients will these drugs be used?

  *   Resistance to HMAs remains an issue for higher-risk patients, and resistance to lenalidomide remains an issue for lower-risk patients. What therapies are being investigated for patients with refractory disease? Which of these therapies will launch between 2012 and 2021 and in which patients will these treatments be used?

  *   Unmet needs in MDS are many and span a range of challenging issues. What unmet needs are expected to remain unaddressed by mid- to late-stage emerging therapies?


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

Primary research: 7 country-specific interviews with MDS, all hematologists.

Epidemiology: Diagnosed incidence segmented by age and country; diagnosed prevalence segmented by morphology classification and level of International Prognostic Scoring System (IPSS) prognostic risk.

Population segments:

- Morphologic classification: refractory anemia (RA), RA with ringed sideroblasts (RARS), MDS with 5q deletion (del[5q]), refractory cytopenia with multilineage dysplasia (RCMD), therapy-related MDS (tMDS), RA with excess blasts (RAEB), RAEB in transformation (RAEB-T), MDS unclassified.

- IPSS classification: low, intermediate-1, intermediate-2, and high; also lower-risk and higher-risk (categories based on the IPSS classifications).

Emerging therapies: Phase I: 8; Phase II: 15 drugs; Phase III: 3 drugs.

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Search Reports

Mentioned in this report:

  • - Accelereon
  • - Actinium
  • - Array Biopharma
  • - Astex
  • - Bioenvision
  • - Celgene
  • - Cell Therapeutics
  • - Cephalon/Teva
  • - Concert
  • - Cornerstone
  • - Cyclacel
  • - Eisai
  • - Eleos
  • - Genentech
  • - Genzyme
  • - GlaxoSmithKline
  • - Incyte
  • - Janssen
  • - MEI Pharma
  • - Merck
  • - MGI Pharma
  • - Novartis
  • - Onconova
  • - Pfizer
  • - Sunesis
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