On July 1, 2025 Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for GLIX1 for the treatment of malignant glioma, a category of devastating brain cancers that includes glioblastoma (Press release, Hemispherian, JUL 1, 2025, View Source [SID1234654200]).

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Significant clinical benefit beyond current therapies.

The designation marks a major regulatory milestone for Hemispherian, recognizing both the urgent unmet medical need in glioma and the potential of GLIX1 to offer significant clinical benefit beyond current therapies.

"This designation from the FDA further validates our scientific approach and mission to address high unmet needs in oncology," said Zeno Albisser, CEO of Hemispherian. "GLIX1 is a first-in-class small molecule with a unique mechanism of action, and we are encouraged by the recognition from both U.S. and EU regulators of its potential to offer meaningful benefit to patients with malignant glioma."

About the Designation

The FDA’s Office of Orphan Products Development granted ODD under section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb). The designation is based on GLIX1’s potential to treat a rare and life-threatening disease affecting fewer than 200,000 people in the United States. GLIX1 demonstrated substantial preclinical efficacy, including tumor regression and survival benefit in validated models of glioma.

Benefits of Orphan Drug Designation

The Orphan Drug Designation from the FDA provides several benefits intended to support and accelerate the development of promising therapies for rare diseases, including:

7 years of market exclusivity in the U.S. upon FDA approval
Tax credits for qualified clinical trial expenses
Exemption from FDA application fees
Eligibility for accelerated regulatory pathways