Hemispherian Receives U.S. FDA Orphan Drug Designation for GLIX1 for the Treatment of Malignant Glioma

Follow Drugslib on

OSLO, Norway, July 1, 2025 /PRNewswire/ -- Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today 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.

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

    • About GLIX1 and Hemispherian

    GLIX1 is a first-in-class small-molecule therapeutic targeting DNA repair vulnerabilities in cancer cells. Hemispherian's proprietary GLIX platform enables the selective elimination of cancer cells with minimal impact on healthy tissue. In preclinical studies, GLIX1 has shown the ability to eradicate tumors in validated animal models with limited side effects, making it a highly promising candidate for the treatment of glioma and other aggressive cancers.

    Hemispherian is an Oslo-based pharmaceutical company focused on developing breakthrough cancer therapies. The company is supported by a growing global network of academic and clinical partners.

    SOURCE Hemispherian AS

    Posted : 2025-07-03 12:00

    Read more

    Disclaimer

    Every effort has been made to ensure that the information provided by Drugslib.com is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Drugslib.com information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Drugslib.com does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Drugslib.com's drug information does not endorse drugs, diagnose patients or recommend therapy. Drugslib.com's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners.

    The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Drugslib.com does not assume any responsibility for any aspect of healthcare administered with the aid of information Drugslib.com provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

    Popular Keywords