ALX Oncology Holdings, an Australian immuno-oncology company focusing on CD47 checkpoint pathway blocking therapies, declared a significant milestone with its flagship drug candidate evorpacept securing the European Commission’s (EC) orphan drug designation (ODD).

This key approval marks a turning point in the treatment for patients suffering from gastric cancer, including gastric cancer and gastroesophageal junction adenocarcinoma (collectively GC).

The critical orphan drug designation approval from the EC follows a similar acknowledgment by the US Food and Drug Administration (FDA) in January 2022, further validating evorpacept as a next-generation solution in the global gastric cancer treatment landscape.

The twin designations highlight evorpacept’s promising potential in delivering effective therapeutic solutions for gastric cancer patients.

Orphan drug designation is a pivotal EC accolade for in-progress medicines intended to treat rare conditions affecting no more than five in 10,000 people within the European Union.

This designation is contingent on either a lack of satisfactory existing treatment options or the new drug demonstrating significant benefit to the affected population.

The orphan drug designation comes with substantial financial and regulatory advantages, such as protocol assistance and possible exemptions or reductions in certain regulatory fees. More significantly, if the designated drug gains marketing approval, it enjoys a decade of market exclusivity in the European Union, paving the way for ALX Oncology’s evorpacept to revolutionize gastric cancer treatment in the European healthcare sector.

Sophia Randolph — ALX Oncology Chief Medical Officer said: “Receiving orphan drug designation from both the EC and the FDA represents a significant regulatory achievement for ALX Oncology and signifies the growing recognition of evorpacept as a potential new drug to improve clinical outcomes in patients with GC.

“Evorpacept has demonstrated promising and consistent anti-cancer activity in the solid tumor setting, and we look forward to presenting data in the second half of 2023 for ASPEN-06, a randomized Phase 2 trial of evorpacept in combination with trastuzumab, paclitaxel and CYRAMZA (ramucirumab) for the treatment of patients with HER2-positive GC.”

  ALX Oncology, Australia, cancer, European Co, European Commission, European Union, evorpacept, gastric cancer, Oncology, Sophia Randolph