Gilead Asks FDA to Rescind Orphan Drug Status for Potential COVID-19 Therapy

Following the approval of orphan drug status for Gilead Sciences Inc’s experimental drug remdesivir, the company has asked the FDA to rescind that designation. However, the drug still has Expanded Access approval.

According to reporting by NPR, the decision may be in response to backlash from the health care community,¹ some of whom said the 7-year exclusivity associated with orphan drug status would limit other companies from developing necessary treatments for the coronavirus disease 2019 (COVID-19).

Orphan drug designation is awarded to investigational drugs for diseases with fewer than 200,000 patients in the United States. As of Thursday, the CDC has confirmed 68,440 cases of COVID-19 in all 50 states, though that number is expected to increase in the coming weeks.²

Agencies such as Knowledge Ecology International (KEI), a non-governmental organization dealing with issues of intellectual property and public health, expressed concern about the orphan drug designation and the process behind obtaining it, specifically that the FDA considered the timeline of submission to be confidential.³

According to a statement from KEI, knowing when the application was submitted is vital to knowing when future companies could begin marketing generic versions.³

“With the date of submission such an absolutely critical piece of data in this case, it is incredible that the FDA would state that this is commercial confidential data,” KEI said in the statement.³

According to NPR, the company had been planning to file a citizen petition against the orphan drug designation, but now won’t have to.¹

Experts are hopeful that remdesivir could be effective against COVID-19 based on its demonstrated activity in animal models against Middle East respiratory syndrome and severe acute respiratory syndrome—both closely related to COVID-19. Remdesivir is a nucleotide analog with broad-spectrum antiviral activity.

In a statement obtained by NPR, Gilead said it plans to continue developing the drug quickly.¹

“Gilead is confident that it can maintain an expedited timeline in seeking regulatory review of remdesivir, without the orphan drug designation,” the statement said. “Recent engagement with regulatory agencies has demonstrated that submissions and review relating to remdesivir for the treatment of COVID-19 are being expedited.”¹

References

• Lupkin S. Gilead Declines ‘Rare Disease’ Status For Experimental Coronavirus Drug. NPR; March 25, 2020. https://www.npr.org/sections/health-shots/2020/03/25/821534016/drugmaker-asks-fda-to-rescind-rare-disease-status-perks-for-covid-19-drug. Accessed March 26, 2020.
• Centers for Disease Control and Prevention. COVID-19 Cases in the US. Updated March 26, 2020. https://www.cdc.gov/coronavirus/2019-ncov/cases-updates/cases-in-us.html. Accessed March 26, 2020.
• Cassedy C. FDA States Submission Date of Gilead’s Coronavirus Treatment Orphan Status Application is ‘Confidential’. Knowledge Ecology International; March 25, 2020. https://www.keionline.org/32581. Accessed March 26, 2020.