Nadunolimab Scores Orphan Drug Designation for Pancreatic Cancer

According to Healio, the FDA recently granted Orphan Drug designation to nadunolimab (CAN04), a treatment candidate for patients with pancreatic cancer. Orphan Drug designation is given to drugs or biologics intended to treat patients with rare conditions, or those affecting under 200,000 Americans. Once a drug receives this status, the drug developer — in this case, biotechnology company Cantargia — also earns a variety of incentives, such as fee waivers, tax credits, increased regulatory assistance, and seven years market exclusivity upon approval. To learn more about Orphan Drug designation, check out this article from the FDA.

Now that nadunolimab has received Orphan Drug designation, it suggests that new and potentially more effective treatments for patients with pancreatic cancer may be on the horizon.


Outside of pancreatic cancer, Cantargia has explored nadunolimab for patients with triple-negative breast cancer. In a press release published on the Cantargia website, researchers explain:

The antibody CAN04 binds strongly to the target IL1RAP and functions both through ADCC as well as blocking IL-1a and IL-1b signaling. Thereby, CAN04 can counteract the contribution of the IL-1 system to the immune suppressive tumor microenvironment.”

In short, nadunolimab binds to the interleukin-1 receptor accessory protein (IL1RAP) to create a more hostile environment for tumors. Through this, researchers believe the treatment can prevent cancer from progressing and reduce tumor size. 

Pancreatic Cancer

Currently, researchers are exploring nadunolimab for pancreatic cancer, cancer that occurs in the pancreas, a hormone-producing organ. There are a number of forms of pancreatic cancer, such as Islet cell tumors and pancreatic adenocarcinoma. 

Patients with pancreatic cancer are often undiagnosed until later stages of the cancer. Once symptoms appear, they include newly-onset or worsening diabetes, unintended weight loss, abdominal and back pain, dark urine, bowel obstructions, appetite loss, fatigue, blood clots, and jaundice. 

In clinical trials, researchers are evaluating the safety, efficacy, and tolerability of this treatment in relation to pancreatic ductal adenocarcinoma. Within the Phase 1/2a CANFOUR clinical trial, researchers are evaluating nadunolimab as a first-line treatment in conjunction with Abraxane. Alternately, the Phase 1B CAPAFOUR clinical trial is exploring nadunolimab in conjunction with FOLFIRINOX. So far, data suggest that treatment helps improve overall and progression-free survival. 

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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