CNS Pharmaceuticals has filed an application with the U.S. Food and Drug Administration (FDA) to receive Orphan Drug Designation (ODD) for its lead product Berubicin.

Under a prior developer, Berubicin, then known as RTA 744, was granted ODD by the FDA for the treatment of malignant gliomas. In the prior developer's Phase 1 trial of Berubicin, 44% of the patients demonstrated a significant improvement in progression free survival. Additionally, one patient in this study experienced a complete response to his treatment with Berubicin.

"We are excited to announce the Orphan Drug application submission for Berubicin, as it would grant special status and accelerate the development of Berubicin to treat glioblastoma, one of the world's most aggressive forms of cancer," said John Climaco, CEO of CNS Pharmaceuticals. "We are pleased to continue to execute upon our strategic initiatives and submit our application within the anticipated timeline outlined within our previous filings. We feel cautiously optimistic about the application, given the past Orphan Drug Designation of the molecule and positive Phase 1 results. We look forward to initiating a Phase II trial evaluating the effect of Berubicin on patients with glioblastoma later this year."

The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation.

The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at any given time. Due to small patient numbers, treatment for these rare diseases would not be considered economically feasible without government programs to support their economic viability. Orphan Drug Designation would qualify Berubicin for certain benefits and incentives, including seven years of marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain activities, eligibility for orphan drug grants, and the waiver of certain administrative fees. The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval.

Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents.  Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata.
 

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