Novel Investigational Product Awarded FDA Orphan Drug Designation Status for the Treatment of Pancreatic Cancer

Novel Investigational Product Awarded FDA Orphan Drug Designation Status for the Treatment of Pancreatic Cancer

The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation status to an investigational drug called Ampligen (rintatolimod) for the treatment of pancreatic cancer, one of the deadliest forms of cancer. Developed by an immuno-pharma company focused on research and development of therapeutics to treat immune disorders, viral diseases, and multiple types of cancers, called AIM ImmunoTech Inc. (NYSE American: AIM), this status offers an orphan status to the investigational product; the status helps incentivize the treatment of therapies to treat unmet medical needs by providing a company with seven years of exclusivity rights once a drug reaches the market.

A Deadly Disease

Pancreatic cancer, the fourth leading cause of cancer deaths in America, is the only cancer among those most commonly diagnosed, with a five-year survival rate at only six percent reports the Pancreatic Cancer Action Network. For more on the facts behind pancreatic cancer see the National Cancer Institute (NCI) website

FDA Orphan Drug Program

The Orphan Drug Designation program provides orphan status to drugs and biologics, which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the United States or meets cost recovery provisions of the act. The status helps incentivize the treatment of therapies to treat unmet medical needs by providing a company with seven years of exclusivity rights once a drug reaches the market.

The Basis for the FDA Action

Recently the drug developer, AIM, announced receipt of statistically significant positive pancreatic cancer survival results from a multi-hear Early Access Program conducted at Erasmus University Medical Center in the Netherlands. That academic medical center, led by Prof. Casper van Eijck, MD, Ph.D., reported that median overall survival was approximately two-fold higher—that is 200%–in the Ampligen arm, as compared to a historic control cohort matched for age, gender, stage of disease and number of cycles of Folfirinox therapy.

According to principal investigator Dr. van Eijck, “The overall survival of the experimental group was compared to a large historical control cohort matched for age, gender, stage of disease and number of cycles of Folfirinox chemotherapy. Medium survival was approximately two-fold higher, that is, 200%, in the Ampligen arm as compared to the historical controls. These results were obtained with a very high degree of statistical significance. Based on these data, I see the potential for Ampligen as a meaningful extension of the standard of care for advanced pancreatic cancer, which we are planning to investigate further.”

About AIM ImmunoTech Inc.

AIM ImmunoTech Inc. is an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19, the disease caused by SARS-CoV-2 virus.

Based in Ocala, Florida, their flagship product Ampligen® (Rintatolimod) is a first-in-class drug of large macromolecular RNA (ribonucleic acid) molecules, and Alferon N Injection® (Interferon Alfa-N3). Ampligen represents an RNA being developed for globally important cancers, viral diseases and disorders of the immune system.

Lead Research/Investigator

Prof. Casper van Eijck, MD, PhD 

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