Immunicum AB (publ) Receives Orphan Drug Designation from EMA for Ilixadencel as Treatment for Gastrointestinal Stromal Tumors (GIST)

Press Release

19 February 2021

Immunicum AB (publ) Receives Orphan Drug Designation from EMA for Ilixadencel as Treatment for Gastrointestinal Stromal Tumors (GIST)

Immunicum AB (publ; IMMU.ST) announced today that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the Company’s application for orphan designation status for its Phase II clinical candidate, ilixadencel, a cell-based, off-the-shelf immune primer, for the treatment of Gastrointestinal Stromal Tumors (GIST). The COMP opinion was based on results from the Phase I/II clinical trial in GIST, a rare and difficult-to treat cancer indication belonging to the group of cancers referred to as soft tissue sarcomas (STS).

“The Orphan Drug Designations awarded by both the EMA and the FDA for ilixadencel in the GIST indication represent an important step forward for Immunicum,” stated Sven Rohmann, M.D., Ph.D., CEO of Immunicum. “They open up an important regulatory pathway and provide us with the potential to rapidly and independently advance ilixadencel toward commercialization in a small patient population. We look forward to executing on our goal of providing new treatment options to GIST patients.” 

To qualify for Orphan Designation in the EU, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU and there must be sufficient data to suggest the investigational medicine may produce clinically relevant outcomes. Applications for orphan designation are examined by the EMA’s COMP, which adopts an opinion that is forwarded to the European Commission. The European Commission’s decision follows a few weeks after the COMP opinion is issued. Orphan drug designation provides companies with certain benefits and incentives, including ten years of market exclusivity upon marketing authorization, clinical protocol assistance, access to a centralized marketing authorization procedure and reduced regulatory fees.

In May 2020, Immunicum received Regenerative Medicine Advanced Therapy designation from the FDA for ilixadencel for the treatment of patients with metastatic Renal Cell Carcinoma. In December 2020, Immunicum announced that it received Fast Track Designation from the FDA for ilixadencel in Gastrointestinal Stromal Tumors (GIST) and Orphan Drug Designation by the FDA for ilixadencel in hepatocellular carcinomas. In January of this year, the Company announced FDA Orphan Drug Designation for ilixadencel in STS.