FDA Grants Enhertu Orphan Drug Designation for Gastric Cancer

WASHINGTON, D.C., US  – The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) for the treatment of patients with gastric cancer, including gastroesophageal junction cancer.

The designation was supported by data from the Phase II DESTINY-Gastric01 trial, in which patients with HER2-positive metastatic gastric or gastroesophageal cancer who were treated with Enhertu, a HER2-directed antibody drug conjugate (ADC), demonstrated a statistically significant and clinically meaningful improvement in objective response rate (ORR). In the trial, the drug also met the primary endpoint, and showed overall survival (OS), a key secondary endpoint, versus patients treated with investigator’s choice of chemotherapy (irinotecan or paclitaxel monotherapy).

AstraZeneca has confirmed that the full results of DESTINY-Gastric-01 will be presented during the 2020 American Society of Clinical Oncology ASCO20 Virtual Scientific Program, 29 to 31 May 2020.

Earlier this month, Enhertu received two Breakthrough Therapy Designations for its potential use in HER2-positive unresectable or metastatic gastric cancer and HER2-mutant metastatic non-small cell lung cancer.

Gastric cancer is the fifth most common cancer worldwide and the third leading cause of cancer mortality with a five-year survival rate of 5% for metastatic disease. In the US, it is estimated that 27,600 new cases of gastric cancer will be diagnosed in 2020 and more than 11,000 people will die from the disease.

The Food and Drug Administration (FDA) grants ODD to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

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