On Monday, Gilead received orphan drug designation for its drug remdesivir, which is being evaluated for the use in COVID-19. However, the manufacturer has now asked the FDA to rescind its orphan drug status for the potential coronavirus therapy.1
Despite this, “Gilead is confident that it can maintain an expedited timeline in seeking regulatory review of remdesivir, without the orphan drug designation," according a statement on its website.1
The orphan designation followed Gilead’s announcement that it is halting emergency access to remdesivir outside of clinical trials for patients with the novel coronavirus disease due to “overwhelming demand.” The manufacturer is now working on transitioning to expanded access programs for patients in need.2
Under orphan drug designation, a new brand-name drug has 7-year exclusivity, which would protect the drug from generic competition. Gilead’s initial decision to seek orphan status for remdesivir quickly drew criticism. Consumer group Public Citizen and other health groups sent a letter on Wednesday to Daniel O’Day, chairman and chief executive officer of Gilead, urging the company to reconsider.3
The Orphan Drug Designation program generally 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. For a drug to qualify for orphan designation, both the drug and the disease or condition must meet certain criteria, according to the FDA. A disease is considered “rare” if it affects less than 200,000 individuals in the United States. Orphan status may also be granted to drugs that meet cost recovery provisions of the act.4
Although Gilead was able to receive orphan drug designation while there were fewer than 200,000 COVID-19 cases in the United States, reported numbers may surpass this as testing continues and the virus spreads. The real number of individuals affected by the disease is unknown, but likely exceeds the number of reported cases.
Gilead is still working on providing access to patients with COVID-19 who may benefit from the investigational therapy. According to Gilead, it is “currently in the process of transitioning the provision of emergency access to remdesivir from individual compassionate use requests to expanded access programs.” Enrollment in clinical trials is now the primary way to access remdesivir; however, expanded access programs will help accelerate access for patients who are severely ill and enable the collection of data from all participating patients. Additionally, Gilead has accelerated manufacturing timelines to increase available supply.2
Gilead has already initiated 2 phase 3 clinical studies to evaluate the safety and efficacy of remdesivir in patients with COVID-19. The studies began enrolling patients in March 2020 and will enroll approximately 1000 patients in the initial phases from countries with high prevalence of the virus.2
“Gilead recognizes the urgent public health needs posed by the COVID-19 pandemic. The company is working to advance the development of remdesivir as quickly as possible, and will provide updates as they become available,” the company said in its statement.1
1. Gilead Sciences Statement on Request to Rescind Remdesivir Orphan Drug Designation [news release]. Gilead’s website. https://www.gilead.com/news-and-press/company-statements/gilead-sciences-statement-on-request-to-rescind-remdesivir-orphan-drug-designation. Accessed March 27, 2020.
2. Emergency Access to Remdesivir Outside of Clinical Trials. Gilead’s website. https://www.gilead.com/purpose/advancing-global-health/covid-19/emergency-access-to-remdesivir-outside-of-clinical-trials. Accessed March 27, 2020.
3. Letter from 50 groups to Gilead renounce remdesivir orphan drug claim. Public Citizen’s website. https://www.citizen.org/wp-content/uploads/Letter-from-50-groups-to-Gilead-renounce-remdesivir-orphan-drug-claim.pdf?eType=EmailBlastContent&eId=f4140734-e5c9-45b8-b67a-d43a461609bf. Accessed March 27, 2020.
4. Designating an Orphan Product: Drugs and Biological Products. FDA’s website. https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products. Accessed March 26, 2020.
5. Remdesivir Clinical Trials. Gilead’s website. https://www.gilead.com/purpose/advancing-global-health/covid-19/remdesivir-clinical-trials. Accessed March 26, 2020.