Pancreatic Cancer Therapy Cambritaxestat Receives FDA Orphan Drug Designation

The FDA has granted Orphan Drug Designation to iOnctura's cambritaxestat, for treating metastatic pancreatic cancer. This designation highlights its unique approach in inhibiting cancer growth, enhancing immune response, and reducing fibrosis.

iOnctura announced that the US Food and Drug Administration (FDA) has awarded its autotaxin inhibitor, cambritaxestat, the Orphan Drug Designation (ODD) for the treatment of pancreatic cancer. This innovative therapy is being studied in combination with chemotherapy to combat metastatic pancreatic cancer. The designation underscores cambritaxestat's potential to significantly improve treatment options, marking a promising step forward for patients.

iOnctura, a clinical-stage biotechnology company, is advancing cambritaxestat, a drug with unique chemical and biological attributes promising safety and efficacy in preclinical studies. Cambritaxestat introduces a groundbreaking approach by targeting autotaxin to inhibit cancer growth, enhance immune responses, and reduce fibrosis. This multifaceted strategy, currently being tested in the Phase I AION-02 trial alongside standard chemotherapy treatments nab-paclitaxel and gemcitabine, aims to revolutionize the treatment of metastatic pancreatic cancer.

Catherine Pickering, CEO of iOnctura, emphasized the significance of the Orphan Drug Designation, stating, "There is an urgent need to develop new therapies for pancreatic cancer which is currently the third largest cause of death by cancer in the U.S., and the fourth in Europe. Although survival of patients with pancreatic cancer has improved in recent years, it still stands at just 13% after five years. This Orphan Drug Designation will support our goal to accelerate cambritaxestat through the clinic to provide a new treatment to patients with limited options."  

Cambritaxestat, recently proposed as the International Nonproprietary Name (INN) by the World Health Organization, represents a groundbreaking therapy for highly fibrotic cancer indications. Notably, it is the first autotaxin inhibitor under investigation for cancer patients.

Recent translational research, published in reputable journals such as ESMO's Immuno-Oncology and Technology, Cancer Research, the Journal of Experimental & Clinical Cancer Research, and Cancers, underscores cambritaxestat's potential in reducing metastasis and tumor outgrowth in preclinical models.

Pancreatic cancer remains a leading cause of cancer-related deaths in the US and Europe, with a five-year survival rate of just 13%. The ODD for cambritaxestat represents a beacon of hope for improving these outcomes.

iOnctura's achievement of Orphan Drug Designation for Cambritaxestat marks a significant stride in advancing novel therapies for pancreatic cancer. This recognition by the FDA underscores the critical impact of iOnctura's innovative approach, offering hope for improved outcomes in a challenging medical landscape.

About Author: Tracy Mokwe

With over 3 years of experience as a medical laboratory scientist and writer, I am passionate about translating complex scientific information into clear and accessible content. My expertise lies in medical sciences, laboratory science and research, digital health, AI, and cybersecurity in healthcare.