On May 18, 2023, Market Watch reported that rucosopasem manganese received Orphan Drug designation. Developed by clinical stage biopharmaceutical company Galera Therapeutics (“Galera”), rucosopasem manganese is largely being developed for individuals living with pancreatic cancer; it is this indication for which the treatment received Orphan Drug designation. Outside of pancreatic cancer, Galera is also exploring rucosopasem manganese for non-small cell lung cancer (NSCLC). 

Orphan Drug designation is granted to therapies that are targeted towards rare conditions affecting fewer than 200,000 nationwide. Galera also receives tax credits, fee waivers, increased FDA communication and assistance, and seven years of market exclusivity if rucosopasem manganese is ever approved. 

The therapy is currently being evaluated in a Phase 2b study in conjunction with stereotactic body radiation therapy. Rucosopasem manganese is described as an investigational selective dismutase mimetic that is infused over a 15-minute period. Its goal is to improve overall survival rates and how effective stereotactic body radiation therapy is. A Phase 1/2 study suggests that the combination treatment is safe and effective and may increase survival rates, shares Galera. 

What is Pancreatic Cancer? 

When you see the name pancreatic cancer, you already know where the cancer forms: the pancreas. Your pancreas has two main functions: to release digestive enzymes and produce hormones that manage your blood sugar. If cancer forms in the pancreatic ducts, it is called pancreatic adenocarcinoma. Pancreatic cancer forming in the neuroendocrine (hormone-producing cells) is more rare; this form is called Islet cell tumor. Being male, smoking, obesity, older age, and a family history of this cancer all increase your risk. 

Symptoms of pancreatic cancer often do not appear until the cancer has progressed, which makes it difficult to diagnose and treat in a timely manner. When symptoms appear, they may include:

• Unintended weight loss
• Blood clots
• Upper abdominal pain that may spread to your back. 
• Appetite loss 
• Itchy skin
• Fatigue
• Jaundice (yellowing of the skin and eyes)
• Newly onset diabetes or diabetes that is becoming more difficult to manage
• Dark urine and pale stools
• Nausea and vomiting
• Enlarged liver and/or gallbladder 
• Blood clots

Right now, the 5-year survival rate of pancreatic cancer is 12%. If you have pancreatic cancer, your doctor may integrate different types of care into your treatment plan: radiation, chemotherapy, targeted therapy, immunotherapy, and surgery. Many clinical studies are also working to identify new treatments that can prolong life.