This approval follows the previous US Food and Drug Administration (FDA) recognitions, including orphan drug and fast track designations awarded in April 2024 and December 2023 to CAN-2409 for pancreatic ductal adenocarcinoma (PDAC).
CAN-2409 is an off-the-shelf, replication-defective adenovirus designed for delivering the herpes simplex virus thymidine kinase (HSV-tk) gene directly to tumour cells.
When used with certain prodrugs (valacyclovir or acyclovir), this HSV-tk enzyme converts the prodrug into DNA-incorporating nucleotide analogues. Thereby it activates a process leading to immunogenic cell death within solid tumours such as prostate cancer and non-small cell lung cancer (NSCLC), alongside PDAC.
Candel president and CEO Paul Peter Tak said: “Receiving EMA Orphan Designation for CAN-2409 represents a significant regulatory milestone for CAN-2409 in this disease. This designation, alongside our existing FDA Orphan Drug and Fast Track Designations for CAN-2409 in PDAC, underscores the promise of our novel multimodal immunotherapy approach.
“The notable benefits we’ve observed with CAN-2409, including evidence of a long tail of survival, highlight the transformative potential of this immunotherapy. As we advance our regulatory strategy across global markets, we remain committed to bringing CAN-2409 to patients who face limited therapeutic options.”
The company reported results from its Phase IIa clinical trial involving CAN-2409 combined with valacyclovir in patients with borderline resectable PDAC.
The data showed an impressive median overall survival rate of 31.4 months when using CAN-2409 with standard care compared to just 12.5 months with standard care alone.
CAN-2409 has received multiple regulatory designations for various solid tumours, including FDA orphan drug and fast track designations for PDAC.
It received additional fast track designations for NSCLC and localised prostate cancer.
Additionally, the therapy holds FDA regenerative medicine advanced therapy (RMAT) designation for intermediate-to-high risk localised prostate cancer.