berubicin获批加速通道指定！多形性胶质母细胞瘤迎来新疗法

Because of its potential to extend overall survival in patients with glioblastoma multiforme, the FDA granted berubicin Fast Track designation for the treatment of this relapsed disease.

According to CNS Pharmaceuticals According to AG, the FDA has granted Fast Track designation to berubicin for the treatment of relapsed glioblastoma multiforme (GBM).

A recently initiated phase 2 study (NCT04762069) evaluated berubicin compared with lomustine in the treatment of recurrent GBM. Previously, the FDA had granted berubicin orphan drug designation.

John, CEO of CNS Pharmaceuticals AG Climaco said in a press release: "We are pleased to receive Fast Track designation from the U.S. FDA for Berubicin, which is a huge achievement for the treatment of glioblastoma, the most aggressive, lethal and treatment-resistant type of cancer that forms in the brain. If there is one disease that requires action to meet clinical need, it is GBM."

Berubicin is a new anthracycline that is the first of its kind to cross the blood-brain barrier. PreviousReata Pharmaceuticals' phase 1 trial demonstrated the drug's anti-tumor activity, including one case of durable complete response.

This open-label, multicenter, two-group parallel trial aims to recruit 210 patients who have failed standard first-line treatment and randomly assign them to the experimental treatment group and the lomustine control group in a 2:1 ratio.

Daily intravenous infusion of berubicin 2 hours, 3 consecutive days, 21 days as a cycle. Lomustine is administered at the approved dose. (Children’s dosage: Orally, 2~3mg/kg each time, once every 6~8 weeks, or 80~100mg/m2 each time, once every 3 weeks. Adult dosage: Orally, 100~130mg/m2 each time, once every 6~8 weeks, 75mg/m2 each time, once every 3 weeks. 3 times is a course of treatment.)

The primary endpoint is overall survival, and secondary endpoints include progression-free survival, event-free survival, and overall response rate.

"Patients have few other options, and thousands of people battle this terrible cancer every year. With Fast Track designation, we have a path to expediting the approval of berubicin, making it easier to bring this therapy to people fighting the disease," Climaco said.

Berubicin The data from the Phase 1 trial are good, with a response rate of 44%, and overall, its safety profile is satisfactory. Currently, the survival rate from glioblastoma diagnosis is only 14.6 months. berubicin can become a new treatment method for this type of patients.

References:

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