FDA grants orphan drug designation to nadunolimab for pancreatic cancer
The FDA granted orphan drug designation to nadunolimab for the treatment of pancreatic cancer.
Nadunolimab (CAN04, Cantargia AB) is an interleukin-1 receptor accessory protein (IL1RAP)-binding antibody in development for various types of cancer.
Two trials are assessing the agent for pancreatic ductal adenocarcinoma, which accounts for more than 90% of pancreatic cancer cases.
The phase 1/phase 2A CANFOUR study is evaluating nadunolimab in combination with gemcitabine and nab-paclitaxel (Abraxane, Bristol Myers Squibb) as first-line treatment. Interim efficacy data from 33 patients showed the regimen led to longer PFS and OS than historical control data.
The phase 1B CAPAFOUR trial is evaluating nadunolimab with FOLFIRINOX as first-line therapy for patients with metastatic pancreatic ductal adenocarcinoma.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.