Treatment for Soft Tissue Sarcoma Receives FDA Orphan Drug Designation

The FDA has granted the cell-based, off-the-shelf immune primer ilixadencel an orphan drug designation for use as a treatment option in patients with soft tissue sarcoma.

The FDA has granted the cell-based, off-the-shelf immune primer ilixadencel an orphan drug designation for use as a treatment option in patients with soft tissue sarcoma (STS), according to an announcement from Immunicum AB.1 The decision was based on findings from a phase 1/2 trial (NCT02432846) evaluating the agent in patients with gastrointestinal stromal tumors (GIST).

When paired with different TKIs, ilixadencel was found to have a promising toxicity profile with no treatment-related serious toxicities observed, meeting the primary safety end point of the trial.2 Moreover, the addition of the agent to a TKI resulted in reduction in tumor size in 2 of 6 patients, despite prior progression on the same TKI. Moreover, 2 of the 6 patients were found to have partial responses (PRs) to the treatment per Choi response criteria; this equated to a 33% objective response rate in this population. One of the 2 patients achieved disease stability for 9 months on third-line regorafenib (Stivarga), while the other had stable disease for 12 months on second-line sunitinib (Sutent). The PRs achieved with the ilixadencel combination suggest that the agent helped to overcome resistance to TKIs in these patients who had experienced progressive disease on prior TKIs.

“Although the GIST study was a trial with few patients enrolled, they were all at an advanced disease stage, meaning that both the safety data and the signals of clinical efficacy are encouraging and support ilixadencel’s potential as a safe and effective cell-based cancer immune primer,” Alex Karlsson-Para, MD, PhD, CSO and interim CEO of Immunicum, stated in a press release. “The publication of our clinical results in a peer reviewed journal further validates the quality of the data generated from our clinical study.”

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