FDA Grants Fast Track Designation to Berubicin For Treatment of Recurrent Glioblastoma Multiforme


Based on its potential to prolong overall survival in patients with glioblastoma multiforme, berubicin was granted fast track designation for the FDA for the treatment of recurrent disease.

Berubicin has been granted fast tracked designation by the FDA for the treatment of glioblastoma multiforme (GBM) following recurrence, according to the company responsible for the agent, CNS Pharmaceuticals AG.1

Patient enrollment was recently initiated for a phase 2 study (NCT04762069) that is investigating the agent versus lomustine chemotherapy as a comparator for the treatment of recurrent GBM. CNS had previously received Orphan Drug Designation from the FDA for berubicin.

“Receiving Fast Track Designation from the U.S. FDA is a huge achievement in our advancement of berubicin for the treatment of glioblastoma, the most aggressive, deadly and treatment-resistant type of cancer that forms in the brain. If there were ever a disease where the unmet clinical need demands action, it is GBM,” John Climaco, chief executive officer of CNS Pharmaceuticals AG, said in a press release.

Berubicin is a novel anthracycline, and appears the first of its kind to cross the blood-brain barrier. This drug has previously demonstrated a antitumor responses including 1 durable complete response in a phase 1 trial by Reata Pharmaceuticals, Inc.2

The open-label, multicenter, 2-arm parallel trial aims to enroll 210 patients following failure of standard first-line therapy who will be randomized in a 2:1 fashion to the experimental treatment arm of berucibin versus active control therapy with lomustine.3 

Daily berucibin will be administered as an intravenous 2-hour infusion for 3 consecutive days of 21-day cycles. Lomustine will be administered through capsules at the institutionally approved dose.

The primary end point is overall survival, with secondary end including progression-free survival, event free survival, and overall response rate.

“Patients have almost no meaningful options and thousands lose their fight against this terrible cancer every year. With this designation, we now have an accelerated pathway to approval for berubicin and a clear opportunity to more expediently bring this potentially impactful investigational therapy to individuals battling this challenging disease,” Climaco said.


1. FDA Grants Fast Track Designation to CNS Pharmaceuticals for Berubicin for the Treatment of Recurrent Glioblastoma Multiforme. News Release. BioSpace. June 29, 2021. Accessed June 30, 2021. https://bit.ly/3jvn61J

2. CNS Pharmaceuticals Berubicin Trial is One Schedule to Commence in March 2021. News Release. Cision. February 25, 2021. Accessed on June 30, 2021. https://prn.to/3h5aqNC

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