Candel Therapeutics Gains EMA Orphan Designation for CAN-2409 in Pancreatic Cancer Treatment
Friday, July 25, 2025
Candel Therapeutics, a clinical-stage biopharmaceutical company, has been granted Orphan Designation by the European Medicines Agency (EMA) for its investigational therapy, CAN-2409 (aglatimagene besadenovec), for the treatment of pancreatic cancer. This status aligns with the therapy's existing designations in the United States, including Orphan Drug and Fast Track status from the U.S. FDA, and highlights the significant need for new treatment options in pancreatic cancer across global markets.
CAN-2409 is an off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene directly into tumour cells. When used alongside an oral prodrug such as valacyclovir, the therapy causes targeted cell death and prompts the release of tumour antigens into the surrounding microenvironment. This process aims to stimulate a systemic immune response, potentially offering benefits across several hard-to-treat solid tumours, including prostate cancer, non-small cell lung cancer (NSCLC), and pancreatic ductal adenocarcinoma (PDAC).
Positive survival outcomes were previously reported from a Phase 2a trial involving CAN-2409 and valacyclovir in patients with borderline resectable PDAC. The trial showed improved overall survival compared to standard treatment alone, with the median survival reaching 31.4 months in the CAN-2409 group versus 12.5 months in the control group. Some patients remained alive over five years after starting treatment, demonstrating encouraging long-term potential.
The EMA’s Orphan Designation supports therapies developed for rare, life-threatening, or chronically disabling conditions affecting fewer than five in 10,000 individuals in the EU. This designation provides benefits such as reduced regulatory fees, scientific guidance, research support, and up to 10 years of market exclusivity in the EU if the product gains approval.
CAN-2409 has received multiple regulatory recognitions in addition to this latest EU designation. It holds FDA Fast Track status for NSCLC and localised prostate cancer, and RMAT (Regenerative Medicine Advanced Therapy) designation in intermediate- to high-risk prostate cancer.
Designed to generate a robust, local and systemic immune response, CAN-2409 combines gene therapy and immunotherapy principles. It works by converting the prodrug into DNA-disrupting agents that cause cell death and inflammation, promoting immune cell infiltration. The result is a targeted immune attack not only on treated tumours but also on distant, untreated metastases.
More than 1,000 patients have received CAN-2409 to date, with the therapy showing a favourable safety profile. Its potential for use in combination with existing standards of care — including radiotherapy, chemotherapy, surgery, and checkpoint inhibitors — further strengthens its clinical appeal across a range of solid tumours.
Source: globenewswire.com
