FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma

Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition

Purdue Pharma has announced a significant milestone in the fight against one of the deadliest brain cancers.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s experimental drug, tinostamustine, for the treatment of malignant gliomas, including aggressive tumors like glioblastoma.

Affecting thousands of patients annually, these cancers have limited treatment options and a grim prognosis, with most glioblastoma patients surviving less than 15 months under current therapies. The FDA’s designation underscores tinostamustine’s potential to address this critical unmet medical need and accelerates its development as a potential first-in-class treatment.

“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,” 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. 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), a global adaptive platform trial for glioblastoma.