FDA Awards Orphan Drug Designation for Brain Cancer Drug Development by Helix 51 Company
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U.P. Oncolytics, a company in Rosalind Franklin University’s Helix 51 biomedical incubator, has been awarded Orphan Drug designation by the FDA’s Office of Orphan Products Development for its oncolytic virus-based therapy to treat malignant glioma.
Orphan diseases are rare conditions that affect fewer than 200,000 people in the U.S. and are often serious or life-threatening. Orphan drug designation is given to drugs showing promise in treating, preventing or diagnosing orphan diseases.
“We are pleased by the FDA Orphan Drug designation,” said U.P. Oncolytics CEO Richard Rovin, MD. “It, along with the SBIR (Small Business Innovation Research) grant, are strong validations of our science and our approach to bring new treatments to this highly resistant cancer.”
RFU announced on Sept. 30 that U.P. Oncolytics had earned a $500,000 NIH award as part of a Phase I-Phase II SBIR Fast-Track grant. The award will fund the company’s proposed in vitro and in vivo IND-enabling studies on the “Validation of a novel treatment for glioblastoma using oncolytic Zika virus.” Aimed at minimizing funding gaps, fast-track grants are awarded to projects that have a high potential for commercialization.
About 80,000 people are newly diagnosed with primary brain tumors each year in the United States. Approximately 25% of those tumors are gliomas. Gliomas form when glial cells grow out of control. They usually grow in the brain but can also form in the spinal cord. Difficult to reach and treat with surgery, they can grow into other areas of the brain. Glioblastoma Multiforme (GBM), the most malignant and pervasive subtype of glioma and the most common primary brain tumor in adults, is included within the scope of the Orphan designation by the FDA.
In 2023, nearly 10,000 people in the United States are estimated to have died from glioblastoma, according to the National Brain Tumor Society. The most common form of malignant brain cancer, glioblastoma accounts for 50% of all malignant brain tumors. The five-year relative survival rate for the disease is 6.9%. Median survival is eight months.
FDA Orphan Drug designation provides numerous benefits to companies, including: market exclusivity for up to 7 years; fast-track review; regulatory guidance; exemption from user fees; and clinical trial expense tax credits.
RFU Interim VP for Research and Dean of the School of Graduate and Postdoctoral Studies Joseph DiMario, PhD, said the orphan designation represents an important milestone in the development of U.P. Oncolytic’s drug candidate.
“U.P. Oncolytics’ progress in developing this novel therapy for GBM brings new hope to patients and their families suffering from this terrible disease,” Dr. DiMario said. “We are greatly encouraged by their development plans and recognition by key regulatory and scientific authorities.”