Orphan Drug Designation granted for treatment of leukaemia  

Blood cancer

Otsuka Pharmaceutical and Astex Pharmaceuticals’ oral fixed dose combination of decitabine and cedazuridine (ASTX727) for the treatment of Acute Myeloid Leukaemia (AML)1 has been granted orphan-drug designation (ODD) by the European Commission (EC). 

What is ALM? 

AML is the most common form of acute leukaemia in adults2. Within Europe, there is an increase in the incidence of AML, which may be attributed to the aging population.  

Official comments  

“The granting of ODD is a key and important step in the journey toward finding a new treatment option for patients with AML”, said Andy Hodge, CEO of Otsuka Pharmaceutical. “We will continue to collaborate with all key stakeholders, including the EC, to make this treatment available to patients in need.” 

Harold Keer, Chief Medical Officer of Astex Pharmaceuticals, said: “AML continues to be a challenging disease area with high rates of relapse and low rates of five-year survival. The disease has a higher incidence rate in people aged over 60 years, which presents an important challenge as the population ages. The granting of ODD signifies that ASTX727 is considered to be a medicine that may potentially benefit those affected by this rare, life-threatening condition.” 


  1. Community Register of orphan medicinal products. European Commission (EC). Available from: https://ec.europa.eu/health/documents/community-register/html/o2548.htm [Last accessed December 2021]. 
  2. De Kouchkovsky I and Abdul-Hay M. ‘Acute myeloid leukemia: a comprehensive review and 2016 update’. Blood Cancer J. 2016;6:e441. 

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