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Ellipses Receives FDA Orphan Drug Designation for RET Inhibitor in Solid Tumor Treatment

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Ellipses Pharma has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for its selective RET inhibitor, EP0031/A400. This drug is being evaluated as a potential treatment for RET fusion-positive solid tumors, which account for approximately 2% of solid tumors and are associated with RET mutations and rearrangements.

In March 2021, Ellipses obtained an exclusive license for EP0031/A400 in Europe and the US from Kelun-Biotech Pharmaceutical while Kelun-Biotech retained specific rights in Greater China. EP0031, developed in collaboration with Kelun-Biotech, is referred to as A400 in regional development efforts with the latter.

Orphan Drug Designation

Promising RET Inhibitor Advances with FDA Orphan Drug Designation

Preclinical trials of EP0031/A400 have demonstrated its effectiveness in inhibiting key RET kinases both in vitro and in vivo. Additionally, it has shown improved penetration of the blood-brain barrier compared to earlier-generation selective RET inhibitors.

The FDA granted investigational new drug application (IND) approval for EP0031/A400 in June 2022, and the orphan drug designation represents a significant milestone in the drug’s development. Ellipses Pharma is now focused on accelerating the development of EP0031 to benefit patients.

In June 2021, China’s National Medicinal Products Administration approved the IND for EP0031/A400, and a registrational Phase II trial is currently underway in China for this therapy.


Resource: Pharmaceutical Technology, November 24, 2023

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