On November 10, 2025 Purdue Pharma L.P. ("Purdue") reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its pipeline drug tinostamustine for the treatment of malignant gliomas, a broad category of brain and spinal cord cancers that affect both adults and children and includes rapidly growing, invasive tumors like glioblastoma. As many as 22,000 people are diagnosed with malignant gliomas annually in the United States1. FDA grants ODD status to encourage development of promising medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

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Purdue is currently investigating tinostamustine in patients with glioblastoma, the most common malignant glioma, that is aggressive, very challenging to treat and for which there is no cure.2 Most patients do not survive more than 15 months with current treatment approaches.3 The ODD request for tinostamustine was supported by robust epidemiologic, clinical and pre-clinical evidence, as well as mechanistic rationale supporting its potential to address a critical unmet medical need for glioblastoma.

"As many as 15,000 people in the U.S. are diagnosed with glioblastoma each year.3 Unfortunately, there is limited survival benefit with existing treatment options," said Julie Ducharme, Vice President and Chief Scientific Officer, Purdue. "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."

Orphan drug designation is intended to facilitate drug development for rare diseases and may provide certain incentives to drug developers.4,5 These benefits include tax credits for qualified clinical trials, exemption from user fees including New Drug Application (NDA), and a potential for seven years of market exclusivity following approval.

Tinostamustine is a potential first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action, bifunctional alkylating activity and pan histone deacetylase inhibition (or HDAC inhibition). Tinostamustine has the potential to be a first-line treatment for glioblastoma.

"Behind every designation like this are real people, patients and families, facing the devastating reality of malignant gliomas, especially glioblastoma," said Craig Landau, MD, President and CEO, Purdue Pharma. "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."

The Company also recently entered into an agreement with the Global Coalition for Adaptive Research (GCAR) to pursue the Phase 2/3 clinical development of tinostamustine in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447), a global adaptive platform trial for glioblastoma.

This press release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that tinostamustine will successfully complete development or gain FDA approval.

(Press release, Purdue Pharma, NOV 10, 2025, View Source [SID1234659749])