On January 13, 2016 Five Prime Therapeutics, Inc. (Nasdaq:FPRX) reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for FPA008 for the treatment of Pigmented Villonodular Synovitis (PVNS), a locally aggressive tumor of the synovium (Press release, Five Prime Therapeutics, JAN 13, 2016, View Source [SID:1234508786]). Schedule your 30 min Free 1stOncology Demo! "PVNS is a painful joint disease for which there are no currently approved treatments," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "FPA008 is our investigational antibody that we believe blocks the activation and survival of the macrophages that make up the bulk of the PVNS tumor. This orphan drug designation by the FDA highlights the need for new treatments to help patients with this rare disease."
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Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to products that treat rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides the sponsor certain benefits and incentives, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.
About PVNS
PVNS is a rare, locally aggressive tumor of the synovium. It is characterized by local over-expression of CSF-1, which recruits macrophages into the joints, forming the non-malignant tumor mass. It is associated with high morbidity, and there are no approved therapies for the condition. FPA008 blocks the binding of CSF-1 and inhibits the activity and survival of the macrophages that form the bulk of the tumor. Five Prime is currently in a Phase 1 clinical trial studying FPA008 as a treatment for PVNS.
About FPA008
FPA008 is an investigational antibody that inhibits CSF1R and has been shown in preclinical models to block the activation and survival of monocytes and macrophages. Inhibition of CSF1R in preclinical models of several cancers reduces the number of immunosuppressive tumor-associated macrophages (TAMs) in the tumor microenvironment, thereby facilitating an immune response against tumors. FPA008 is currently in phase 1 clinical trials in PVNS and oncology indications. FPA008 is being developed under an exclusive worldwide license and collaboration agreement with Bristol-Myers Squibb (BMS).