FDA Grants Orphan Drug Designation to Rintatolimod for Pancreatic Cancer

December 23, 2020 - The FDA has granted an orphan drug designation to the mismatched, double-stranded RNA molecule rintatolimod for the treatment of patients with pancreatic cancer.

The FDA has granted an orphan drug designation to the mismatched, double-stranded RNA molecule rintatolimod (Ampligen)for the treatment of patients with pancreatic cancer, according to an announcement from AIM ImmunoTech, Inc.1

The decision followed results from a multiyear Early Access Program held at Erasmus University Medical Center, which indicated that the agent resulted in a statistically significant overall survival (OS) benefit in this patient population. Notably, the median OS with rintatolimod was approximately double that, 200% higher, of the historical control cohort that had been matched for age, gender, disease stage, and number of cycles of FOLFIRINOX (folinic acid, fluorouracil, irinotecan hydrochloride, and oxaliplatin) treatment.

“These study data demonstrate that [rintatolimod] has the potential to extend the survival rates of people suffering with pancreatic cancer significantly when compared with the traditional standard of care for this deadly disease,” Thomas K. Equels, CEO of AIM ImmunoTech, Inc, stated in a press release.

Results from a single-center pilot cohort study evaluating rintatolimod as maintenance treatment in patients with pancreatic cancer showed that the agent was able to elicit an immune response and shift neutrophil-to-lymphocyte ratio (NLR) and elevated systemic inflammation index (SII) in a beneficial way.2

Elevated NLR and SII are associated with poor survival outcomes in patients with pancreatic cancer. In the pilot study, investigators set out to evaluate whether rintatolimod was able to stimulate changes in the NLR and SII in this patient population; they also wanted to further evaluate systemic immune profile changes.

Patients with any stage of pancreatic cancer were enrolled to the study. Following standard-of-care treatment, patients were administered maintenance rintatolimod through a patient program. Throughout the study, investigators evaluated the NLR ratios, SIIs, and progressive disease in patients who received the treatment. Blood samples were collected and assessed via flow cytometry and a broad panel of immune cells associated with TLR3 were also measured.

A total of 26 patients were included in the analysis and 65.4% of these patients were male with a mean age of 62.3 years. Following 6 weeks of treatment with rintatolimod, the NLR was found to be substantially elevated in the 8 patients who experienced disease progression (P = .029) vs the 11 patients who did not progress. Over the 18-week timeframe, a drop in NLR and SII was observed in participants who did not experience progressive disease, while an increase in NLR and SII was noted in those who did.

The orphan drug designation program offers orphan status to agents and biologics that are considered to be intended for the treatment, prevention, or diagnosis of a rare disease or condition that impacts fewer than 200,000 individuals in the United States or meets cost recovery provisions of the act. The status helps to encourage the development of therapies to address unmet medical needs by giving companies 7 years of exclusivity rights once the drug reaches the market.

The company is also working with its Contract Research Organization, Amarex Clinical Research LLC, to also obtain fast track and potentially breakthrough designations from the FDA, along with investigational new drug authorizations to launch a follow-up phase 2/3 trial to further examine the agent.3


  1. AIM ImmunoTech Inc.’s drug Ampligen awarded FDA’s orphan drug designation status for the treatment of pancreatic cancer. News release. AIM ImmunoTech Inc. December 21, 2020. Accessed December 23, 2020. http://bit.ly/3pozTmB.
  2. Latifi D, Moskie M, Mustafa D, et al. Rintatolimod (Ampligen) maintenance therapy in pancreatic cancer patients: results from a single center pilot cohort study. HPB. 2018;20(suppl 2):S570. doi:10.1016/j.jpb.2018.06.2049
  3. AIM ImmunoTech receives statistically significant positive survival results in pancreatic cancer from Erasmus University Medical Center, Rotterdam, Netherlands. News release. AIM Immunotech, Inc. September 22, 2020. Accessed December 23, 2020. http://bit.ly/3nJBdjm.