Ambrx, a clinical stage biopharmaceutical company using an expanded genetic code to create Engineered Precision Biologics, announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for ARX788 for the treatment of patients with HER2-positive gastric cancer, including cancer at the gastroesophageal junction.
The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation for ARX788 for gastric cancer provides Ambrx with certain benefits, including an exemption to FDA prescription drug user fees and tax credits for qualified clinical trials. Orphan drug designation also confers eligibility for seven years of market exclusivity to an orphan drug post-approval, subject to receiving marketing approval from the FDA.
“The ongoing Phase 1 ACE-Gastric-01 trial has shown promising anti-tumor activity in HER2-positive advanced gastric patients who have been previously treated with trastuzumab and chemotherapy in the metastatic setting,” said Feng Tian, Ph.D., President and CEO of Ambrx. “Receiving orphan drug designation from the FDA is an important milestone in our ongoing efforts to develop ARX788 for a wide range of HER2-positive cancers. We are proud to be targeting a rare disease that is currently severely underserved by advancing ARX788 into additional clinical trials.”
The company anticipates the release of additional Phase 1 data from the ACE-Gastric-01 trial by the end of 2021 and initiation of ACE-Gastric-02, a global Phase 3 trial for HER2-positive gastric cancer, in the second half of 2021. ACE-Gastric-02 is currently planned to be a randomized trial of ARX788 versus physician’s choice of treatment in second line HER2-positive gastric cancer and HER2-positive cancer at the gastroesophageal junction.