Ellipses Pharma's EP0042 Granted Orphan Drug Designation by FDA for AML Treatment

Ellipses Pharma obtains Orphan Drug Designation (ODD) from the FDA for EP0042, a dual FLT-3 and Aurora kinase inhibitor, as a treatment for acute myeloid leukaemia (AML). This designation is granted to promising investigational treatments for rare diseases, like AML, affecting fewer than 200,000 people in the US. In February 2023, the FDA approved EP0042’s Investigational New Drug Application, enabling additional trial sites in the US for this compound.

Publish Date: 14-03-2023 Source: Ellipses Pharma

Acute myeloid leukemia (AML) is the most common type of adult leukemia, but it has 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 a 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). It has been estimated that approximately 22,850 adults will be diagnosed with AML in 2020; it accounts for the highest percentage (62%) of leukemic deaths.

However, the current Acute myeloid leukemia (AML) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Azacitidine, Decitabine, Midostaurin, etc..) has 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 the Acute myeloid leukemia treatment. The key companies in the advanced development stage are AbbVie, Eisai Inc., Novartis Pharmaceuticals, etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast 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 2021 to 2032.

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