FDA Grants Orphan Drug Status to Lisata's Certepetide for Cholangiocarcinoma

Lisata Therapeutics, Inc. (Nasdaq: LSTA), a company specializing in the development of innovative treatments for advanced solid tumors and other severe diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its investigational drug, certepetide, for the treatment of cholangiocarcinoma. This rare and aggressive form of cancer affects the bile ducts and has limited treatment options, making it a significant challenge for patients, particularly after initial therapies fail.

Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata, emphasized the importance of this designation, highlighting it as a critical milestone in addressing the unmet needs of cholangiocarcinoma patients. The orphan status will help propel certepetide through the clinical development stages, ultimately aiming to bring new treatment options to those affected by this challenging cancer.

Lisata is actively testing certepetide in its BOLSTER trial, a Phase 2a study designed to assess the drug's effectiveness in treating first- and second-line cholangiocarcinoma. This trial is a double-blind, placebo-controlled, multi-center, randomized study being conducted in the United States.

The FDA's Orphan Drug Designation is a significant advantage for drugs aimed at treating rare conditions, defined as those affecting fewer than 200,000 people in the U.S. Benefits of this designation include up to seven years of exclusive marketing rights post-approval, exemption from certain user fees, and eligibility for tax credits on qualified clinical trials. Additionally, it often results in expedited clinical development through closer cooperation with the FDA.

Cholangiocarcinoma, or bile duct cancer, is notoriously difficult to diagnose accurately and is often misclassified. The American Cancer Society estimates approximately 8,000 new cases in the U.S. annually, although the actual number is likely higher due to diagnostic difficulties. The condition has a poor prognosis, with a five-year survival rate of less than 5%, underscoring the urgent need for new treatment solutions.

Certepetide operates by selectively activating the C-end rule active transport mechanism in tumors, thereby enhancing the penetration and accumulation of co-administered anti-cancer drugs in the tumor tissue. It also modifies the tumor microenvironment to reduce its immunosuppressive characteristics and inhibit metastasis. Non-clinical data have shown that certepetide improves the delivery and efficacy of various anti-cancer therapies, including chemotherapies, immunotherapies, and RNA-based treatments. The drug has demonstrated promising safety, tolerability, and clinical activity in ongoing trials, which aim to prove its ability to boost the effectiveness of standard-of-care chemotherapy for pancreatic cancer as well as combined chemotherapy and immunotherapy for various solid tumors.

Certepetide has already received several designations, including Fast Track and Orphan Drug status for pancreatic cancer in both the U.S. and E.U., and Orphan Drug status for glioma, osteosarcoma, and cholangiocarcinoma in the U.S. It has also been awarded Rare Pediatric Disease Designation for osteosarcoma in the U.S.

Lisata Therapeutics remains committed to discovering and developing breakthrough treatments for severe conditions. The company's strategic focus is on leveraging its CendR Platform® technology to enhance the delivery and effectiveness of anti-cancer drugs. Lisata has established significant partnerships and anticipates several key milestones in the coming 18 months, with operations expected to be funded into early 2026.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!