The U.S. Food and Drug Administration (FDA) has given orphan drug designation to Provectus Pharmaceuticals, Inc., for Rose Bengal, the active component in their new oncology medication PV-10. The drug is designed for the treatment of hepatocellular carcinoma (HCC), the most prevalent form of liver cancer. At present Provectus is designing a Phase II investigation, following the January 2011 completion of their Phase I study, which involved patient accrual and treatment of PV-10 for liver cancer in all participants.
Since Provectus was the first company to receive marketing approval for this drug, the FDA has given them orphan drug designation which entitles Provectus to exclusive marketing rights for the liver cancer PV-10 drug in the U.S. for up to 7 years, in addition to a waiver of certain user fees required by the Prescribing Drug User Fee Act (PDUFA).
Dr. Craig Dees, PhD, CEO of Provectus, explained:
"Receiving orphan drug designation for hepatocellular carcinoma, in combination with our strategy for patent protection of PV-10, is another important step in assuring the value of the company's proprietary property once regulatory approval is achieved. Based upon our clinical trials of PV-10 for metastatic melanoma, as well as on the results we are seeing in our Compassionate Use Program for PV-10 for non-visceral cancers, we believe PV-10 will be a safe and effective treatment for many different types of cancers. Preliminary analysis of the results of the Phase 1 liver cancer trial shows that PV-10 should have a role in the treatment of tumors of the liver.
Orphan drug designation for liver cancer will provide us market exclusivity, as well as financial and regulatory benefits, provided that the trials are completed successfully and the treatment receives FDA approval."
About PV-10PV-10 is a proprietary, injectable formulation of Rose Bengal, a compound that has been used for over three decades by ophthalmologists to evaluate eye damage. In addition, it has been used as an intravenous diagnostic to identify ailments of the liver. It has an established safety history, a short half-life in the bloodstream, and is discharged through the liver and kidneys. Based on the observation Rose Bengal is selectively toxic to cancer calls via a process called chemoablation whereby cells undergo a form of cell death that mimics both features of necrosis and apoptosis, Provectus has found a new use for Rose Bengal.
Provectus has completed its Phase II clinical study of PV-10 for metastatic melanoma, and at present they are in conversation with both the Food and Drug Administration (FDA) regarding the design of a pivotal Phase III randomized controlled trial suitable for SPA ("Special Protocol Assessment"), and the Australian Therapeutic Goods Administration to review regulatory approval of PV-10 for melanoma in Australia.
Written by Grace Rattue