By Jaimy Lee
The Food and Drug Administration’s decision to grant a full approval to Gilead Sciences Inc.’s remdesivir is raising questions about the broader value of a drug that has been praised for being “first” but may only provide limited clinical benefits.
Shares of Gilead /zigman2/quotes/210293917/composite GILD +0.42% gained 4.1% in trading on Friday, the day after the FDA announced the approval of Veklury, the brand-name for remdesivir. It’s the first COVID-19 treatment to receive an FDA approval during the pandemic.
“The balance of the data shows that it is providing a benefit,” Dr. Scott Gottlieb, a former FDA commissioner, told Squawk Box Friday morning. “We have to be comfortable with singles and doubles when it comes to developing drugs against COVID right now. We rushed a lot of medicines into clinical development, not necessarily optimizing them. This is sort of the first generation of medicines. Nothing, probably, is going to be a home run.”
The FDA in May granted a closely watched emergency use authorization to remdesivir, a move that sent markets up the following week over hopes that new treatments would slow down the pace of deaths and hospitalizations associated with COVID-19 infections. (An EUA is a less rigorous regulatory authorization that is being used during the public-health emergency to speed up the process of bringing new medications to coronavirus patients.)
The basis of the Veklury authorization was the antiviral drug’s ability to help hospitalized patients recover faster, which is beneficial for both patients and the hospitals caring for those patients. Since May, additional research has backed this up, and the U.S. government has launched several new trials testing other drugs in combination with Veklury, now that it is considered the “standard of care” for hospitalized COVID-19 patients in the U.S.
Yet other studies, including two trials in China and one conducted by the World Health Organization, have found little benefit in Veklury. The WHO’s Solidarity trial is a randomized, open-label study evaluating four antiviral drugs, including Veklury, in 11,330 hospitalized patients around the world; it found that the drugs had “little or no effect” on mortality, the need for ventilation or duration of hospital stay, according to interim findings published Oct. 15 in preprint form, meaning they haven’t been peer-reviewed. Gilead contests these findings.
“While the Solidarity trial provided information about Veklury in real-world settings in the early days of the pandemic, it does not negate other study results,” Dr. Merdad Parsey, Gilead’s chief medical officer, said in a statement announcing the FDA approval.
At the same time, some medical experts are questioning the basis for the approval and have also wondered why the FDA did not hold an advisory committee meeting to discuss the risks and benefits of the drug. In the traditional FDA approval process, a group of independent clinical experts convenes for a public meeting to discuss experimental therapies before a FDA decision. They vote on whether the agency should approve a drug. The FDA isn’t required to follow the committee’s recommendation, but it often does.
“Not supportive of this decision at all,” Dr. Eric Topol, a cardiologist and director of the Scripps Research Translational Institute, tweeted Thursday. “Does it work early? Does it work late? Does it work anytime? So much unresolved.”
Dr. Peter Bach, an epidemiologist and director of Memorial Sloan Kettering Cancer Center’s Center for Health Policy and Outcomes, called the approval “astonishing” in a tweet , before criticizing the use of clinical trials that either lacked a control arm or blinding in the approval. “An entirely negative real-world [randomized controlled trial] was larger than these put together,” Bach tweeted. “‘Effective’? What?”
(Three three trials cited by the FDA in total enrolled about 7,000 participants. The WHO study has about 11,000.)
The FDA’s label for the drug “surprisingly” excludes findings from the two trials conducted in China and the WHO Solidarity trial, according to Raymond James analyst Steven Seedhouse. “In other words, the label excludes negative trials/data for remdesivir,” he wrote in a note to investors.
Separately, the emergence of the mortality data from the WHO trial has prompted the Institute for Clinical and Economic Review, a drug-pricing watchdog, to revise its cost-effectiveness assessment of Veklury. Data from that model is expected to be published in the next two weeks, according to an ICER spokesperson. Back in July, ICER had said that without a “clear mortality benefit,” Gilead should lower the price of the drug. Veklury currently costs $3,120 for a course of treatment.
After the initial rush of support in May, Wall Street has largely been ambivalent about Veklury’s potential impact on Gilead’s stock performance. The company’s stock is down 6.6% for the year, compared with the S&P 500 /zigman2/quotes/210599714/realtime SPX +0.88% , which has gained 6.9%.