27 April 2020 - - US-based biotechnology company CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) has filed an application with the US Food and Drug Administration to receive Orphan Drug Designation for its lead product Berubicin, the company said.

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.

The Orphan Drug Act 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 utilise natural processes to induce deoxyribonucleic acid 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.

CNS Pharmaceuticals is a biotechnology company specializing in the development of novel treatments for primary and metastatic brain and central nervous system tumors.

Its lead candidate Berubicin is proposed for the treatment of glioblastoma, a type of brain cancer currently considered incurable, as well as for pancreatic and ovarian cancers, and lymphomas.

The company entered into an intellectual property agreement with Houston Pharmaceuticals, Inc. and a Purchase Agreement with Reata.