On February 24, 2021 AIM ImmunoTech Inc. (NYSE American: AIM) reported that its subsidiary, NV Hemispherx Biopharma Europe, reported that it has received formal notification from the European Commission (EC) approving the company’s Orphan Medicinal Product Application for Ampligen as a treatment for pancreatic cancer (Press release, AIM ImmunoTech, FEB 24, 2021, View Source [SID1234575530]).

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Medications that have been designated as Orphan products by the European Medicines Agency (EMA), once commercially approved in the European Union (EU), receive benefits including up to ten years of protection from market competition from similar medicines with similar active component and indication for use that are not shown to be clinically superior.

AIM announced earlier this month that the Committee for Orphan Medicinal Products of the EMA had recommended that Ampligen receive the designation for pancreatic cancer. The company has now received the official approval of that designation.

Prof. Casper van Eijck, M.D. Ph.D., the lead investigator for the expanded access program (EAP) for Ampligen at Erasmus Medical Center, stated, "We are pleased to report that the EMA has approved Ampligen for orphan medicinal product designation. Pancreatic cancer is the seventh leading cause of cancer-related deaths worldwide with over 458,000 worldwide cases of pancreatic cancer in 2018 alone. Despite advancements in the detection and management of pancreatic cancer, the 5-year survival rate is only 5-10%. Due to the positive and statistically significant survival results, versus historical controls, that we observed when using Ampligen in patients with locally advanced/metastatic pancreatic cancer-after systemic chemotherapy-we believe that Ampligen has the potential to be a meaningful extension of the standard of care for advanced pancreatic cancer. We are currently writing the manuscript describing the results of our study and are planning to investigate Ampligen further in the follow-up pancreatic cancer Phase 2/3 clinical trial and in a Phase 1 trial in combination with check point inhibitors at Erasmus Medical Center."

Kazem Kazempour, Ph.D., at Amarex Clinical Research, commented, "We are working closely with AIM in the United States to attain FDA ‘fast-track’ status, as well as possible FDA ‘breakthrough’ designations, and to obtain IND authorizations to conduct a follow-up pancreatic cancer Phase 2/3 clinical trial with sites in the Netherlands at Erasmus MC under Prof. van Eijck, and also at major cancer research centers in the United States."

"Orphan medicinal product designation in the EU and in the US is a critical milestone in AIM’s ongoing efforts to develop Ampligen as a treatment for pancreatic cancer," said AIM CEO Thomas K. Equels. "Pancreatic cancer is one of the deadliest cancers because, far too often, it is not diagnosed until Stage IV, when the disease is so far along that there are limited therapeutic options. We at AIM hope that Ampligen can one day help add precious time to the lives of many people suffering from pancreatic cancer."

Orphan medicinal product designations promote the clinical development of drugs that target rare life-threatening conditions, and which are expected to provide significant therapeutic advantage over existing treatments. An estimated 466,000 people died of pancreatic cancer worldwide in 2020, according to the World Health Organization. The five-year survival rate is only 5-10 percent.

Sponsors who obtain EMA orphan designation "benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market," according to the EMA. Fee reductions are also available depending on circumstances. The EMA has a comprehensive explanation of the benefits of Orphan Drug Designation (ODD) on its website.

The EMA designation follows a similar approval from the U.S. Food and Drug Administration (FDA), which also awarded AIM with orphan drug designation status for Ampligen as a treatment for pancreatic cancer.

The FDA orphan designation followed the company’s September 22, 2020 announcement of statistically significant positive pancreatic cancer survival benefit in the Ampligen arm as compared to a historical control cohort seen in a multi-year Early Access Program conducted at Erasmus University Medical Center in the Netherlands. The use of Ampligen following the current standard of care for pancreatic cancer (FOLFIRINOX) yielded an overall survival of 19 months, 7.9 months greater than FOLFIRINOX treatment alone.