The FDA has recently granted orphan drug designation to Ezurpimtrostat, a PPT-1 inhibitor for the treatment of patients with hepatocellular carcinoma (HCC).
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"Orphan Drug"
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Data from a study recently published by the Royal Marsden Cancer Centre showed promising data for the combination of Avutometinib + Defactinib in the treatment of ovarian cancer, resulting in significant tumour shrinkage in 28% of patients and disease control in 93% of patients.
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Apparatus
FDA Grants Orphan Drug Status To Two New Therapies In a Row, And These Two Groups Of Patients Are Expected To Benefit!
Recently, the US FDA has granted orphan drug status to two new therapeutics in a row. One is a small molecule inhibitor, PCLX-001, for the treatment of acute myeloid leukaemia; the other is a T-cell therapy, ET140203, for the treatment of hepatoblastoma. Both therapies are currently in relevant trials.