The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to tinostamustine, a treatment candidate from Purdue Pharma that aims to extend survival for people with malignant gliomas, the developer announced.
Gliomas are a group of tumors that form from glial cells — those that support and protect the nerve cells in the brain and spinal cord — and can be benign, or noncancerous, or malignant, where they grow and spread. Few treatments are now available for malignant gliomas.
The FDA designation is awarded to help speed development of treatments for rare diseases, which, in the U.S., are those that affect fewer than 200,000 people. It provides benefits such as tax credits for clinical trials, waiver from certain fees, and up to seven years of market exclusivity if a therapy is ultimately approved.
According to Julie Ducharme, PhD, Purdue’s vice president and chief scientific officer, tinostamustine “has shown promise in early trials” testing the medication.
“This recognition from [the] FDA is an important milestone in our mission of advancing innovative science in areas of serious, unmet medical need,” Ducharme said in a company press release. “We look forward to further investigating tinostamustine.”
Tinostamustine now being tested in global clinical trial
Among patients, some malignant gliomas, such as glioblastoma, can grow and spread rapidly, invading and destroying neighboring healthy tissue.
Tinostamustine is a first-in-class medication that works through two mechanisms simultaneously: It damages the DNA of cancer cells while blocking histone deacetylases, enzymes crucial for cancer cell growth. This increases its potential to be more effective against glioblastoma, a disease that remains difficult to treat, according to the company.
“Behind every designation like this are real people, patients and families, facing the devastating reality of malignant gliomas, especially glioblastoma,” said Craig Landau, MD, Purdue’s president and CEO. “Tinostamustine represents a step forward in our efforts to help address the urgent and unmet needs of those affected by these aggressive cancers.”
Tinostamustine has recently been included in GBM AGILE (NCT03970447), a Phase 2/3 clinical trial designed to speed the testing of new treatments for glioblastoma.
GBM Agile, run with the Global Coalition for Adaptive Research, employs a flexible design that allows it to test multiple treatments simultaneously and adjust based on early results. Its goal is to identify those therapies that extend survival. The trial is now enrolling patients in Australia, Europe, and North America.
Behind every designation like this are real people, patients and families, facing the devastating reality of malignant gliomas, especially glioblastoma. … Tinostamustine represents a step forward in our efforts to help address the urgent and unmet needs of those affected by these aggressive cancers.
“As many as 15,000 people in the U.S. are diagnosed with glioblastoma each year,” Ducharme said. “Unfortunately, there is limited survival benefit with existing treatment options.”
Ducharme said she looks forward to further investigating tinostamustine after positive results in earlier trials. According to Purdue, benefits have been seen in both preclinical and clinical data.
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