Gilead Rescinds Orphan Drug Request

Biopharmaceutical producer Gilead Sciences has surprised the market by asking the US Food and Drug Administration (FDA) to rescind the orphan drug status of its experimental antiviral drug remdesivir only a day after the application was granted.

Remedesivir had previously received positive mention in the New England Journal of Medicine, which reported that it had been successfully used on a first patient infected by COVID-19.  Gilead is currently conducting two international trials with the drug that had earlier failed as a potential Ebola treatment, and the National Institutes of Health (NIH is also running a US trial.

The granting of orphan status provoked a backlash from US public health agencies and consumer advocates, however, not least because of the benefits the drugmaker would have received, in particular allowing it to charge higher prices.

Orphan drug designation is intended to spur development of treatments for rare diseases by awarding the manufacturer tax breaks, FDA fee waivers and seven years without generic competitors. For these reasons, these drugs are among the most expensive.

To gain orphan status, a drugmaker must show its product serves a population of fewer than 200,000 people in the US. On the day of the FDA approval there were just over 60,000 confirmed cases of COVID-19 across the country, but the number was expected to surge past the 200,000 mark.

Among critics of the FDA’s move, non-profit public interest group Knowledge Ecology International said “it's embarrassing to take something that's potentially the most widespread disease in the history of the pharmaceutical industry and claim it's a rare disease."

But even without the orphan drug status, Gilead said it is confident it can maintain an expedited timeline in seeking regulatory review, noting that “recent engagement with regulatory agencies has demonstrated that submissions and review relating to remdesivir for the treatment of COVID-19 are being expedited."