
Jul 18 2025
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European Commission has designated oral decitabine and cedazuridine as orphan drugs for the treatment of Acute myeloid leukemia (AML)
Astex Pharmaceuticals, Inc. today announces that the European Commission (EC) has granted orphan-drug designation to the oral fixed-dose combination of decitabine and cedazuridine for the treatment of Acute Myeloid Leukemia (AML). ODD-granted therapies, such as ASTX727, entitle companies to 10 years of market exclusivity once the product is approved, among other benefits.
Source: Astex Pharmaceuticals, Inc.
Acute myeloid leukemia (AML) is the most common type of leukemia in adults yet continues to have the lowest survival rate of all leukemias. Although rates have improved remarkably in the younger age group, the prognosis in older patients continues to be very poor. Chemotherapeutic agents, such as alkylating agents and topoisomerase II inhibitors, have been reported to increase the incidence of AML. Several other substances have been linked to an increased risk of AML. Chronic exposure to certain chemicals clearly shows an increased risk for the development of AML. Benzene is the best-studied and most widely used potentially leukemogenic agent.
- AML is a malignant disorder of the bone marrow characterized by the clonal expansion and differentiation arrest of myeloid progenitor cells. The age-adjusted incidence of AML is 4.3 per 100,000 annually in the United States (US).
However, the current Acute myeloid leukemia (AML) treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (PLX3397, TL-895, gilteritinib, AZD1152, ALX148, Dociparastat sodium, etc.) have been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.
In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for Acute myeloid leukemia (AML) treatment. The key companies in the advanced development stage are Plexxikon, Telios Pharma, Inc., Astellas Pharma Global Development, Inc., AstraZeneca, ALX Oncology Inc., etc.
Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook report on Acute myeloid leukemia (AML) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2020 to 2030.
Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.
Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making
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