Propanc Biopharma has submitted an orphan drug designation request to the U.S. Food and Drug Administration (FDA) for PRP, its lead product for treatment of ovarian cancer.
PRP is an intravenous therapy composed by two pancreatic proenzymes – trypsinogen and chymotrypsinogen.
“Obtaining orphan drug designation from the FDA for our PRP therapy for ovarian cancer is a significant regulatory milestone that we are looking forward to, and will be a positive step forward in Propanc Biopharma’s ongoing efforts to develop effective treatments for metastatic cancer,” James Nathanielsz, Propanc Biopharma’s CEO, said in a press release.
Previous preclinical studies testing PRP’s effects in several cancer stem cells and mouse models of ovarian cancer suggested that the potential therapy halted a process where epithelial cells acquire motile and invasive characteristics typical of mesenchymal cells. Scientists believe that this process – known as epithelial-mesenchymal transition – allows cancer cells to leave their primary location and travel into distant locations within the body, increasing their likelihood to generate metastasis.
By halting epithelial-mesenchymal transition, PRP could stop tumor progression and metastasis formation in ovarian cancer. Moreover, additional results suggest it also could repress the development of cancer stem cells (CSCs).
Some scientists suggest that cancer stem cells help cancer cells to resist current cancer therapies, including chemotherapy. This leads to cancer recurrence, experienced by many ovarian cancer patients, which remains one of the main challenges for today’s therapies.
The preliminary data supporting PRP’s effects in cancer stem cells and epithelial-mesenchymal transition grants PRP with high hopes for improving current ovarian cancer therapies.
The Orphan Drug Act was set up to encourage the development of drugs for rare diseases. If granted, drug developers qualify for a seven-year FDA-administered market Orphan Drug Exclusivity (ODE), and for a series of financial incentives (clinical trial tax incentives and waived FDA fees, e.g.). They also receive assistance for designing clinical trial protocols.
“This will further reinforce our strategic investment in PRP, since we already achieved ODD [orphan drug designation] status for pancreatic cancer, demonstrating significant progress in developing a potential best in class therapy that could transform treatment for patients with metastatic cancers, where limited treatment options are available,” said Nathanielsz. “At the time the ODD is granted, we expect to be working closely with the regulatory authorities and clinical trial investigators to advance PRP promptly through the next stages of clinical development, ultimately filing a clinical trial application for first-in-human studies in the UK early next year,” he said.