FDA Grants Berubicin Orphan Drug Status for Brain Cancer

The FDA has granted an orphan drug designation to berubicin for the treatment of patients with malignant gliomas.

The FDA has granted an orphan drug designation to berubicin for the treatment of patients with malignant gliomas, according to CNS Pharmaceuticals, Inc., the manufacturer of the anthracycline.1

“We are pleased to receive Orphan Drug designation for berubicin, our lead candidate. The designation provides berubicin with a special status that can accelerate its development to treat malignant gliomas, and provides CNS with the potential for market exclusivity upon the drug's approval," John Climaco, CEO of CNS Pharmaceuticals, stated in a press release.

"In the phase 1 trial of berubicin to treat glioblastoma, one of the world's most aggressive cancers, under a prior developer, 44% of the patients demonstrated a significant improvement in progression-free survival, and one patient experienced a complete response. We look forward to continuing to execute on our strategic plan and initiating a phase 2 trial evaluating the effect of berubicin on patients with glioblastoma later this year," added Climaco.

The purpose of the FDA Office of Orphan Products Development (OOPD) is to facilitate the development and review of treatments that show potential to improve treatment for patients with rare diseases or conditions. In this line, the OOPD grants an Orphan Drug designation to drugs and biologics that are being developed for the treatment, diagnosis, or prevention of rare diseases/disorders, which the OOPD defines as those affected fewer than 200,000 people in the United States.

"We are excited to continue to drive the development of berubicin and work towards addressing a critical unmet medical need. Glioblastoma currently has a dismal survival rate of only 14.6 months from its diagnosis. We believe berubicin, which based on limited clinical data appears to be the first anthracycline to cross over the blood brain barrier in adults, provides a potentially novel therapy for the treatment of malignant gliomas," Sandra Silberman, MD, chief medical officer of CNS Pharmaceuticals, stated in a press release.

CNS Pharmaceuticals plans to launch a phase 2 clinical trial by the end of the year that will explored berubicin as a treatment for patients with glioblastoma multiforme.

According to the Nation Cancer Institute, the estimated number of new cases of brain and other nervous system cancers in 2020 is 23,890, which represents 1.3% of all newly diagnosed cancers.2 The estimated numbers of deaths this year due to brain and other nervous system cancers is 18,020. Based on the most recent estimate, spanning 2010 to 2016, the 5-year relative survival rate for patients with these cancers is 32.6%. The most recent (2017) estimate available from the National Cancer Institute stated that there are over 168,494 individuals in the United States living with brain and other nervous system cancers


  1. CNS Announces the FDA has Granted Orphan Drug Designation for Brain Cancer Drug Berubicin. Published June 11, 2020. https://prn.to/2UBdBkv. Accessed June 11, 2020.
  2. Cancer Stat Facts: Brain and Other Nervous System Cancer. NIH National Cancer Institute Surveillance, Epidemiology, and End Results Program. https://bit.ly/2UB5u7r. Accessed June 11, 2020.