Hemispherian Gains EU Orphan Drug Status for GLIX1 in Glioma

Hemispherian AS, a leading biotech company based in Oslo, Norway, made an exciting announcement on June 6, 2025. The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has given a positive nod, recommending Orphan Drug Designation (ODD) for Hemispherian's pioneering treatment, GLIX1. This molecule is targeted at glioma—a highly aggressive and lethal brain cancer.

Receiving this designation is a landmark achievement for Hemispherian, marking a critical step in the regulatory process. It highlights the severe unmet medical needs associated with glioma and the promising potential of GLIX1 to provide substantial clinical benefits beyond what current treatments offer. Zeno Albisser, CEO of Hemispherian, expressed pride in the recognition from EMA, stating it as a validation of their scientific approach and a bolster to their mission of revolutionizing glioma treatment. Patients currently face very limited and largely ineffective treatment options, making advancements like these crucial.

The COMP conducted a thorough assessment and concluded that GLIX1 fulfills the criteria for orphan designation under Regulation (EC) No 141/2000. Glioma is a life-threatening and chronically debilitating condition, affecting about 2.6 individuals per 10,000 people in the European Union. Presently available therapies for Glioblastoma, a type of glioma, provide minimal survival benefit, with median overall survival usually less than 15 months. GLIX1 has demonstrated significant tumor reduction and extended survival in validated animal models, even achieving complete tumor eradication in some cases. This suggests that GLIX1 has a clinically significant advantage over existing treatments, meeting the EMA's criteria for a "significant benefit."

The Orphan Drug Designation carries substantial benefits for Hemispherian. These include accelerated market access and ten years of market exclusivity in the EU following approval. Additionally, Hemispherian will receive protocol assistance and regulatory guidance from EMA during clinical development and be eligible for fee reductions on regulatory submissions, including marketing authorization. These incentives are part of efforts to spur the development of innovative treatments for rare diseases with high unmet needs.

Hemispherian is a pharmaceutical company based in Oslo, focused on creating a new class of small-molecule drugs aimed at treating glioblastoma and other aggressive cancers. Their proprietary GLIX platform uses cutting-edge DNA-targeting technology designed to enhance patient survival and quality of life. So far, GLIX1 has demonstrated impressive effects with minimal to no side effects, and crucially, it does not harm healthy tissue.

The designation of GLIX1 as an Orphan Drug is a significant milestone in Hemispherian's journey to develop effective treatments for glioma. This achievement not only acknowledges the potential of GLIX1 but also the urgent need for new therapies that could change the treatment landscape for glioma patients. With the backing of EMA, Hemispherian is poised to make substantial advancements in the field of oncology, offering hope to those affected by one of the most challenging cancers.