Following a public backlash to Monday’s news that the FDA had granted Gilead Sciences Inc. an orphan drug designation for remdesivir, an antiviral in development to treat COVID-19, the Foster City, Calif., company is taking the unprecedented step of rescinding its request for the designation.
Along with giving up the orphan designation, Gilead said it’s waiving all benefits that come with it, including tax credits, seven years of market exclusivity and a waiver of the requirement to provide a pediatric study plan prior to submitting a new drug application. That requirement could slow the rush to get a new drug approved in an emergency situation, as it can take up to 210 days to review a pediatric study plan, according to the company.
In announcing its decision Wednesday, March 25, Gilead said it’s “confident that it can maintain an expedited timeline in seeking regulatory review of remdesivir without the orphan drug designation.” Its recent engagement with regulators has demonstrated that submissions and review relating to remdesivir as a COVID-19 treatment are being expedited, it added.
“Gilead recognizes the urgent public health needs posed by the COVID-19 pandemic,” it said. “The company is working to advance the development of remdesivir as quickly as possible.”
Gilead requested the orphan drug designation early this month when about 500 COVID-19 infections had been confirmed in the U.S. and about 110,000 cases had been reported worldwide. The orphan drug status is granted to drugs that are being developed to treat diseases affecting fewer than 200,000 patients in the U.S.
When the FDA granted the designation this week, there were more than 43,600 COVID-19 cases in the U.S. And as of today, the World Health Organization is reporting more than 413,000 cases in 197 countries, with 18,433 deaths worldwide. While the number of cases in the U.S. would still qualify remdesivir for orphan drug status, the fear is that the number could escalate into the millions – thus the backlash.
Much of that backlash came from politicians, some of whom blamed the Trump administration and the FDA for granting orphan drug status to remdesivir. Sen. Bernie Sanders (I-Vt.), who’s contending to be the Democratic presidential candidate, chided the FDA for “exploiting a law reserved for rare diseases to privatize a drug to treat a pandemic virus.” Besides calling on the administration to rescind what he called a corporate giveaway, Sanders said the administration should make any COVID-19 treatment and vaccine free for everybody.
“Now is not the time for profiteering in the pharmaceutical industry. Now is the time to bring our scientists together to develop and produce the best treatment for the coronavirus as quickly as possible,” Sanders said.
‘Very unique situation’
When BioWorld asked the FDA this week about its decision to grant orphan drug status to remdesivir, an agency spokesperson focused on efforts by the FDA and Gilead to provide emergency access to the investigational new drug (IND).
Noting that, in addition to clinical trials, Gilead has provided emergency access to the drug for severely ill patients in Europe, Japan and the U.S., the spokesperson said the company “is currently transitioning the provision of expanded access for remdesivir from an individual emergency IND mechanism to an expanded access protocol.” The broad expanded access program will be less burdensome to providers and patients and is expected to accelerate the delivery of the drug to hospitals, physicians and patients, according to the agency.
What’s left unanswered in all this is whether the Orphan Drug Act requires the FDA to consider an orphan drug request based on the current number of people with a specific disease or on the potential or expected growth of those numbers.
“This is definitely a very unique situation,” Chad Landmon, chair of the intellectual property and FDA practice groups at Axinn, Veltrop & Harkrider LLP, told BioWorld. “Most [orphan] diseases for which therapies are being developed have been around for years, so there’s not an exponential growth going on.”
Gilead isn’t the only biopharma giving up market exclusivities in the time of COVID-19. Mylan NV announced Wednesday that it’s voluntarily waiving the 180-day market exclusivity for the first generic to Abbvie Inc.’s HIV drug, Kaletra (lopinavir/ritonavir). Mylan said it was taking the action to help increase the available supply of the drug should it prove effective in the treatment of the coronavirus, as other generics would be able to come to market.
Kaletra didn’t meet its primary endpoint, time to improvement, in a recent 200-patient randomized trial that was among the first to try to repurpose an approved drug for COVID-19.