FDA Grants Orphan Drug Status to Tempest's TPST-1495 for FAP Treatment

In a recent announcement, Tempest Therapeutics, Inc., a clinical-stage biotechnology firm, shared exciting news regarding the progress of one of its innovative treatments. The U.S. Food and Drug Administration (FDA) has recognized TPST-1495, a dual receptor inhibitor developed by the company, with an Orphan Drug Designation (ODD). This novel therapeutic aims to address Familial Adenomatous Polyposis (FAP), a rare genetic condition predisposing individuals to cancer. The designation is a pivotal moment for Tempest, marking significant progress in their mission to develop groundbreaking treatments for conditions with limited medical options.

Stephen Brady, the President and CEO of Tempest Therapeutics, expressed the importance of this recognition. He emphasized that achieving ODD status for TPST-1495 is a crucial milestone in their pursuit to deliver innovative solutions for patients who have unmet needs. This latest designation highlights the company's commitment to advancing therapies that can significantly impact patients' lives, building upon previous recognitions they have received for other treatments, like amezalpat in the context of hepatocellular carcinoma.

The company plans to initiate a Phase 2 clinical trial of TPST-1495 for patients with FAP within the year. This study will be conducted through the Cancer Prevention Clinical Trials Network, with funding provided by the National Cancer Institute's Division of Cancer Prevention. The results from this trial are anticipated to be available in 2026, potentially paving the way for new treatment options for FAP patients.

Familial Adenomatous Polyposis (FAP) is an inherited syndrome that significantly increases the risk of cancer in affected individuals. In the United States, it is estimated that around 1 in 5,000 to 10,000 people are impacted by this condition. FAP results from mutations in the APC gene, a tumor suppressor, and leads to the formation of numerous adenomatous polyps throughout the gastrointestinal tract. These growths typically appear during adolescence and carry a high potential for cancerous transformation, affecting the colon, stomach, duodenum, rectum, and other areas. The primary treatment for FAP involves early surgical removal of the colon, known as a colectomy, to decrease cancer risk. However, even after surgery, patients require ongoing monitoring for cancer elsewhere in the gastrointestinal tract throughout their lives. Although surgical interventions and surveillance have improved patient outcomes, cancer remains a significant cause of mortality in those with FAP.

The FDA's Orphan Drug Designation is a program designed to incentivize the development of treatments for rare diseases, affecting fewer than 200,000 people in the U.S. This designation provides various benefits to the companies, such as tax credits for clinical testing, exemption from certain FDA application fees, and a period of seven years of market exclusivity if the drug receives approval.

Tempest Therapeutics is headquartered in Brisbane, California, and is dedicated to advancing a diverse array of small molecule treatments. Their product candidates focus on tumor-targeted and immune-mediated mechanisms with the potential to treat a broad spectrum of tumors. The company's innovative programs are in various stages of development, ranging from initial research to advanced studies in global trials for first-line cancer patients.