FDA Grants Orphan Drug Designation for Brain Cancer Drug Berubicin

CNS Pharmaceuticals Inc., a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, have announced that the FDA has granted Orphan Drug Designation (ODD) for its lead product Berubicin for the treatment of malignant gliomas.

"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," stated John Climaco, CEO of CNS Pharmaceuticals. "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 II trial evaluating the effect of Berubicin on patients with glioblastoma later this year."

Chief medical officer of CNS, Dr. Sandra Silberman, stated, "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."

The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.