Designation follows recent U.S. Breakthrough Therapy Designations for ENHERTU for HER2 positive metastatic gastric cancer and HER2 mutant metastatic non-small cell lung cancer
Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and AstraZeneca’s ENHERTU® (fam-trastuzumab deruxtecan-nxki) has been granted Orphan Drug Designation (ODD) in the U.S. for the treatment of patients with gastric cancer, including gastroesophageal junction cancer.
The U.S. Food and Drug Administration (FDA) grants ODD to medicines intended for the treatment, diagnosis or prevention of rare diseases of disorders that affect fewer than 200,000 people in the U.S.
An estimated 27,600 new cases of gastric cancer will be diagnosed this year, and the disease could lead to more than 11,000 deaths in the U.S. in 2020.1
The phase 2 DESTINY-Gastric01 trial demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of objective response rate (ORR) and the secondary endpoint of overall survival (OS) for patients with HER2 positive metastatic gastric or gastroesophageal cancer treated with ENHERTU, a HER2 directed antibody drug conjugate (ADC), versus physician’s choice of chemotherapy (irinotecan or paclitaxel monotherapy).
The overall safety and tolerability profile of ENHERTU in DESTINY-Gastric01 was consistent with that seen in the phase 1 gastric cancer trial in which the most common adverse events (≥30%, any grade) were hematologic and gastrointestinal including neutrophil count decrease, anemia, nausea and decreased appetite. There were cases of drug-related interstitial lung disease (ILD) and pneumonitis, the majority of which were grade 1 and 2 with two grade 3 and one grade 4. No ILD-related deaths (grade 5) occurred in patients with gastric cancer in the phase 1 trial or in the DESTINY-Gastric01 trial.
The research results of DESTINY-Gastric01 will be presented at the 2020 American Society of Clinical Oncology (ASCO20) Virtual Scientific Program.
Earlier this month, ENHERTU received two Breakthrough Therapy Designations from the FDA for the treatment of patients with HER2 positive unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma who have received two or more prior regimens including trastuzumab, and for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 mutation and with disease progression on or after platinum-based therapy.
ENHERTU also received SAKIGAKE designation in March 2018 from Japan’s Ministry of Health, Labour and Welfare (MHLW) for potential use in HER2 positive gastric cancer, and a supplemental New Drug Application was recently submitted to the Japan MHLW.
