Myelodysplastic Syndromes | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2017

Myelodysplastic syndromes ( MDS ) is a heterogeneous collection of hematopoietic stem cell disorders that result in cytopenias and risk of progression to acute myeloid leukemia ( AML ). Management of anemia through erythropoiesis-stimulating agents or more aggressive treatment with hypomethylating agents has long been the standard of care for lower- and higher-risk MDS patients, respectively. Development of therapies for patients who become refractory to standard-of-care treatments is the key unmet need for this market. The MDS pipeline is robust, and several projected market entrants are poised to penetrate subpopulations of the lower-risk MDS market. While development of novel agents for higher-risk MDS patients is active, this segment of the market has proved to be challenging for developers to demonstrate overall survival benefit. 
Questions Answered: 

  • How do interviewed experts perceive currently available MDS therapies? 
  • Will further research into genetic markers impact either diagnosis or prognosis in MDS ? 
  • What emerging therapies are most likely to impact the MDS market in 2026?  
  • How will Celgene’s oral azacitidine ( CC -486) be received by physicians? What will be the off-label use if the label is restricted to lower-risk patients? 
  • Will Celgene’s luspatercept ( ACE -536) be limited to a second-line option for lower-risk MDS patients? 
  • What therapies currently marketed or are in late-phase development for AML will have relevance in the MDS market? 


Markets covered : United States, France, Germany, Italy, Spain, and the United Kingdom. 
Primary research : Six country-specific interviews with thought leading hematologists. 
Epidemiology : Incident and diagnosed prevalent cases of MDS by country, morphology, and prognostic risk; including drug-treated cases. 
Market forecast : Drug-level sales and patient share of key MDS therapies in 2026. 
Emerging therapies : Phase III: 7 drugs. Phase II: 21 drugs. Coverage of select Phase I/II and I products. 
Key companies : Celgene, Otsuka, Amgen, Janssen, Acceleron, Novartis, Agios, Jazz, Celator, Onconova, Astex. 
Key drugs : Azacitidine (Vidaza, generics), decitabine (Dacogen, generics), Revlimid (lenalidomide), epoetin alfa (Epogen, Procrit), darbepoetin alfa (Aranesp), CC -486, luspatercept, eltrombopag (Promacta/Revolade), enasidenib, CPX-351 (Vyxeos), rigosertib (Estybon), guadecitabine. 

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