RARE Daily

FDA Grants Gilead Orphan Drug Designation for Experimental COVID-19 Treatment

March 24, 2020

The U.S. Food and Drug Administration has granted Gilead Sciences Orphan Drug designation for remdesivir, its experimental treatment in late-stage trials for the treatment of COVID-19, the coronavirus at the heart of the current global pandemic.

The designation has engendered outrage because it will give Gilead a 7-year monopoly on marketing the drug if and when it gains FDA approval, and potentially jack up its price. Under provisions of the Orphan Drug Act, the government will be unable to contract with a company to produce a generic until the exclusivity period ends because it will not be approved for marketing.

The FDA grants Orphan drug designation to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act filing fees.

The Orphan Drug Act of 1983 defines a rare disease as one affecting fewer than 200,000 people in the United States. In the European Union, legislation introduced in 2000 defines rare disease as a disorder affecting fewer than one in 2,000 people.

As of March 23, when the designation was granted, the Centers for Disease Control and Prevention had confirmed more than 33,000 cases through testing, with 400 deaths.

But the novel coronavirus is not expected to remain rare as it is highly infectious and humans have no innate immunity to it. What happens when more tests become available and incidence surpasses 200,000 people, or .06 percent of the U.S. population?

According to Jamie Love, director of the watchdog non-profit Knowledge Ecology International (KEI), Gilead’s decision to seek Orphan Status will rely on a provision in U.S. law that states that determination of the designation “with respect to any drug shall be made on the basis of the facts and circumstances as of the date of the request for designation of the drug.”

KEI has also written a brief identifying the role the U.S. Army, CDC, and the National Institutes of Health have played in the early preclinical development of remdesivir, which began after the Ebola virus outbreak in West Africa in 2014.

In late February, the NIH’s National Institute of Allergy and Infectious Diseases began a randomized, controlled clinical trial to evaluate the safety and efficacy of remdesivir in hospitalized adults diagnosed with COVID19.


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