Hemispherian AS announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation for GLIX1, the company's lead therapeutic candidate for treating glioma. The Oslo-based biotech company's milestone represents a significant regulatory advancement for addressing one of the most devastating and lethal brain cancers.
Regulatory Milestone Validates Scientific Approach
The COMP determined that GLIX1 meets the criteria for orphan designation under Regulation (EC) No 141/2000 following a detailed review. The designation recognizes both the urgent unmet medical need in glioma and the potential of GLIX1 to offer significant clinical benefit beyond current therapies.
"We are proud to have received this recommendation for an Orphan Drug Designation from the EMA Committee. This validates our scientific approach and supports our mission to transform treatment for patients with glioma, who currently face extremely limited and ineffective options," said Zeno Albisser, CEO of Hemispherian.
Addressing Critical Unmet Medical Need
Glioma affects approximately 2.6 in 10,000 people in the EU and represents a life-threatening and chronically debilitating disease. Current treatment options demonstrate significant limitations, with existing therapies for glioblastoma offering only limited survival benefits and median overall survival typically less than 15 months.
Promising Preclinical Results
Non-clinical studies with GLIX1 demonstrated significant tumor reduction and extended survival in validated animal models, including cases of complete tumor eradication. These results suggest that GLIX1 offers a clinically relevant advantage over existing treatments, fulfilling the EMA's criteria for "significant benefit."
The company's proprietary GLIX platform utilizes unique DNA-targeting technology aimed at improving patient survival and quality of life. According to Hemispherian, the treatment shows impressive effects with limited to no side effects and no damage to healthy tissue.
Commercial and Development Incentives
The Orphan Drug Designation provides Hemispherian with substantial development and commercial advantages, including 10 years of market exclusivity in the EU upon approval. Additional benefits include protocol assistance and regulatory guidance from EMA during clinical development, along with eligibility for fee reductions for regulatory submissions, including marketing authorization.
These incentives are specifically designed to encourage the development of innovative treatments for rare diseases with high unmet need, supporting Hemispherian's mission to develop next-generation therapeutics for aggressive cancers through their novel class of small-molecule drugs targeting glioblastoma and other aggressive cancers.
