Tempest Therapeutics announced that the FDA granted Orphan Drug Designation to TPST-1495 for treating Familial Adenomatous Polyposis (FAP). TPST-1495 is a novel dual receptor inhibitor of prostaglandin (PGE2) signaling, and this marks the company’s second clinical program to receive this designation. A Phase 2 study, funded by the National Cancer Institute, is scheduled to begin this year with results expected in 2026.
This FDA designation is important because FAP is a rare, inherited syndrome with no approved medical therapies, affecting approximately 1 in 5,000 to 10,000 individuals in the US. Currently, the standard of care involves surgical removal of the colon, often early in life, followed by lifelong monitoring for cancer development in other areas of the GI tract. The Orphan Drug Designation highlights the unmet need in this patient population and the potential for TPST-1495 to offer a much-needed non-surgical treatment option.
The Orphan Drug Designation provides Tempest with several benefits, including tax credits for clinical testing, potential fee waivers for FDA applications, and seven years of market exclusivity if TPST-1495 is ultimately approved. The Phase 2 trial, conducted by the Cancer Prevention Clinical Trials Network, will provide crucial data regarding the efficacy and safety of the drug in FAP patients.
This development represents a significant step forward for Tempest Therapeutics and for individuals with FAP. Positive results from the upcoming Phase 2 trial could pave the way for a new treatment paradigm for this high-risk population, potentially delaying or even eliminating the need for drastic surgical intervention. This progress underscores the potential of targeted therapies like TPST-1495 to address unmet medical needs in oncology.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
