A new mesothelioma treatment targeting the Hippo signaling pathway has been granted Orphan Drug Designation from the U.S. Food and Drug Administration. The decision means that Orion Pharma will receive special tax credits, user fee exemptions, and eligibility for seven-year market exclusivity for its investigational drug.
Mesothelioma Drug ODM-212 Targets Hippo Pathway Dysfunction
ODM-212 is an oral, small-molecule pan-TEAD (Transcriptional Enhanced Associate Domain) inhibitor being tested in a Phase 2 clinical study for the treatment of pleural mesothelioma, as well as epithelioid hemangioendothelioma and other solid tumors with Hippo pathway dysfunction. The trial includes mesothelioma patients who have progressed after receiving standard treatments and who have no further treatment options.
ODM-212 works by targeting the Hippo signaling pathway in mesothelioma. The pathway is known to regulate cell growth and organ size, but dysregulation — particularly through YAP/TAZ activation—can lead to uncontrolled tumor growth and resistance to cancer therapies. ODM-212 blocks TEAD transcription factors, disrupting YAP-TEAD protein-protein interactions and inhibiting TEAD auto-palmitoylation, both of which are essential for TEAD activity and mesothelioma tumor proliferation.
Clinical Trial Evaluates Safety and Response Rates in Mesothelioma Patients
The global trial assessing ODM-212’s impact on mesothelioma is being conducted at leading oncology centers in the United States and Europe, with primary endpoints including safety and tolerability and secondary endpoints measuring Overall Response Rate, Progression Free Survival, and Overall Survival. These are critical metrics for evaluating whether ODM-212 can extend life and improve outcomes for mesothelioma patients who have exhausted standard treatment options.
“Receiving orphan drug designation for mesothelioma is an important milestone for the ODM-212 program,” said Dr. Praveen Aanur, Chief Medical Officer of Oncology Therapy Area at Nordic-based Orion Pharma. “It underscores the importance of developing urgently needed innovative therapies for patients living with mesothelioma.”
The FDA grants orphan drug designation to investigational therapies addressing rare diseases or conditions affecting fewer than 200,000 people in the U.S., including mesothelioma. With approximately 3,300 Americans diagnosed annually, this aggressive asbestos-related cancer typically has a poor prognosis and limited treatment options.
Clinical trials and investigational drugs hold the promise of a better future for those diagnosed with mesothelioma. If you or someone you love has been diagnosed with this rare, asbestos-related disease, the Patient Advocates at Mesothelioma.net are here to help. Contact us today at 1-800-692-8608 to learn more.
