Can You Rescind an Orphan Designation? Lessons from Gilead-FDA
​​[Thursday, March 26, 2020]
This week Gilead’s decision to ask FDA to rescind its orphan drug designation for its COVID-19 drug highlighted the loophole in the overall rules for orphan drug designations. Unlike all other expedited approval designations, orphan drugs designations (ODD), once granted by the FDA cannot be rescinded by the FDA, under almost all circumstances. Gilead reportedly filed its ODD request in early March and received the designation practically in weeks, a very fast turnaround for FDA for a designation request particularly when one would think FDA was focused on dealing with a rapidly evolving pandemic. Gilead’s application was very well-timed as it took incidence rate numbers early on in the pandemic where they were below the threshold of 200,000 in the US (thankfully the numbers are still below 200,000 at time of this writing), and it was timed where FDA wants to do everything to incentivize development of a therapy. This should emphasize to all concerned the importance of early filing for expedited approval designations. Although FDA can rescind other expedited approval designations such as breakthrough, Fast Track, Accelerated Approval, it rarely does. However, early requesters have a higher chance of getting a designation than the applicants who come after several similar designations have been granted by the FDA. It is even harder for FDA to revoke an ODD; once granted, FDA cannot take the ODD back even if the incidence rate increase per the law. The only allowed reasons for revocation of an ODD are if the applicant lied or hid relevant information in its ODD request. Coming back to the case of the Gilead ODD for its coronavirus drug Remdesivir, the request to rescind the ODD by Gilead is unique. Gilead has no legal reason to do so and FDA cannot ask it to withdraw. So, we are left to the generosity of Gilead to not use its ODD to block all competition to the generic for two additional years to the 5 years market exclusivity it will get anyway. Either way, the incidence created huge publicity for Remdesivir as a treatment of coronavirus even though the trials to test the drug have not yielded any results so far. It primes the Remdesivir market for coronavirus way before its launch (if the clinical trials succeed). It shows how a skilled regulatory team can create a win-win situation for the company, no matter what the outcome of a development program. Kudos to the Gilead regulatory team to demonstrate to all regulatory teams how this should be done.