FDA Grants Drug Designation to Tinostamustine for Malignant Glioma

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to tinostamustine, an investigational therapeutic from Purdue Pharma L.P., for the treatment of patients with malignant gliomas, according to a news release.

Orphan drug designation is designed to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S. This status provides significant incentives for developers, including tax credits for qualified clinical trials, exemptions from certain FDA fees and up to seven years of market exclusivity once approved, the release explained.

“Behind every designation like this are real people, patients and families, facing the devastating reality of malignant gliomas, especially glioblastoma,” Dr. Craig Landau, president and CEO of Purdue Pharma, stated. “We are deeply committed to pursuing this medicine that has the potential to bring hope where few options exist today. Tinostamustine represents a step forward in our efforts to help address the urgent and unmet needs of those affected by these aggressive cancers.”

For patients and families, this designation may represent a new option for therapies in areas of critical medical need, where current treatment options remain extremely limited.

Tinostamustine’s Potential in Glioblastoma Treatment

Purdue is actively investigating tinostamustine in patients with glioblastoma, the most common and aggressive form of malignant glioma. Glioblastoma is a cancer that originates in the brain or spinal cord. It grows rapidly and can infiltrate and damage surrounding healthy tissue, according to the Mayo Clinic website, which added that this type of tumor develops from astrocytes, the cells that support nerve cells. Malignant glioma can affect both adults and children, the news release adds, noting that approximately 22,000 people in the United States are diagnosed with malignant gliomas each year.

Glioblastoma is highly invasive, grows rapidly and is extremely difficult to treat with existing therapies, which provide only limited survival benefit. Most patients with glioblastoma survive fewer than 15 months under current treatment approaches, the news release explained. The FDA’s orphan drug designation was supported by extensive clinical and preclinical evidence, suggesting that tinostamustine has the potential to address this urgent unmet medical need.

Tinostamustine is a potential first-in-class therapy, representing a new chemical entity with two complementary mechanisms of action. It combines bifunctional alkylating activity with pan histone deacetylase (HDAC) inhibition, a dual approach that may increase the drug’s effectiveness against tumor cells. This combination could potentially serve as a first-line therapy for patients newly diagnosed with glioblastoma.

Global Glioma Clinical Trials Expand Access and Research

Purdue has also partnered with the Global Coalition for Adaptive Research (GCAR) to advance tinostamustine in the GBM AGILE trial (Glioblastoma Adaptive Global Innovative Learning Environment), a global adaptive platform study. This innovative trial design allows multiple therapies to be tested efficiently while adapting to emerging results, potentially accelerating the development of promising therapies for glioblastoma.

For patients, participation in GBM AGILE may provide early access to new treatments while contributing to vital research that could improve outcomes for future patients.

“As many as 15,000 people in the U.S. are diagnosed with glioblastoma each year. Unfortunately, there is limited survival benefit with existing treatment options,” Julie Ducharme, vice president and chief scientific officer of Purdue, said in the news release. “This recognition from FDA is an important milestone in our mission of advancing innovative science in areas of serious, unmet medical need. We look forward to further investigating tinostamustine, which has shown promise in early trials.”

Reference

1. “FDA Grants Orphan Drug Designation for Tinostamustine in Malignant Glioma,” by Purdue Pharma L.P. (“Purdue”). News release; Nov. 10, 2025.
2. “Glioblastoma,” by the Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/glioblastoma/symptoms-causes/syc-20569077

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