Immunicum AB announced via news release that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ilixadencel, an off-the-shelf cell-based cancer immunotherapy aimed to treat gastrointestinal stromal tumors (GISTs).

“We continue to interact with agencies on the development of treatments for hard-to-treat tumors,” Jeroen Rovers, MD, PhD, chief medical officer of Immunicum, said.

“This [Orphan Drug Designation] will support further development of ilixadencel, including a Phase II trial which Immunicum is currently preparing with ilixadencel in combination with tyrosine kinase inhibitors in GIST.”

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The FDA granted Orphan Drug Designation status to ilixadencel for the treatment of soft tissue sarcoma in January 2021 and Fast Track Designation to the drug for GIST in December 2020. Thus far, ilixadencel has been evaluated in several clinical trials on solid tumors such as metastatic renal carcinoma and hepatocellular carcinoma in addition to GIST.

The treatment has also been tested in combination with standard cancer therapies including the tyrosine kinase inhibitors, sunitinib and regorafenib, and the checkpoint inhibitor, pembrolizumab.

With its demonstrated efficacy and encouraging safety and tolerability profile, Immunicum plans to focus on assessing the use of ilixadencel in combination with tyrosine kinase inhibitors as therapy in patients with GISTs.


Immunicum AB: Immunicum received FDA Orphan Drug Designation for ilixadencel as treatment of gastrointestinal stromal tumors (GIST). News release. Immunicum AB; June 9, 2022.