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5 FDA approval decisions to watch in the 4th quarter

The next three months will be among the most consequential in the Food and Drug Administration’s modern history.

This fall, at least two of the leading coronavirus vaccine makers could seek emergency approvals, setting up what will be a series of momentous decisions for the regulator. The FDA will face immense pressure to clear a vaccine, but will be forced to make a call with significantly less information than usual to go on, due to the extraordinary speed at which developers have proceeded.

Should the agency authorize an experimental vaccine for emergency use, or grant a full approval, tens of millions of people will likely receive a shot, even under a controlled roll-out to high-risk groups.

Further complicating the agency’s task is a U.S. presidential election, in which the Trump administration’s response to the COVID-19 pandemic will be a top issue among voters. President Donald Trump has staked much to obtaining a vaccine before Nov. 3, publicly pressuring the FDA with timelines that are at odds with those of his own public health officials.

Amid all of this, the FDA will also be carrying out its usual business. Approval decisions are scheduled for a number of important drugs, including a cancer cell therapy for lymphoma, a combination drug for schizophrenia, a new anemia pill and an antibody treatment for the Ebola virus.

The five experimental therapies are listed in order of the currently scheduled date by which the FDA has agreed to make a decision on approval. There are no scheduled review times for SARS-CoV-2 vaccines. However, the FDA is widely expected to consider granting emergency authorizations prior to a standard review.

Vaccines for SARS-CoV-2 infection

Four drugmakers are testing would-be vaccines for the new coronavirus in large, late-stage trials in the U.S. Two, Pfizer and Moderna, expect to get preliminary data from their studies by, respectively, late October and November.

It’s not clear exactly what any early results would look like, but detailed study plans made public by the companies give some sense of how the trials are being conducted and when they might be declared a success (or failure).

Each trial has enrolled tens of thousands of healthy adults, randomizing them to receive either a placebo or the experimental shot being studied. Relatively few cases of COVID-19 will be needed to determine whether vaccination is protective — 164 in Pfizer’s trial and 151 in Moderna’s.

But the drugmakers have built interim data checks into their trials, allowing independent monitoring committees to review results after as few as 32 and 53 cases, respectively. In either case, the vaccine would need to be highly effective, or clearly ineffective, for the committee to reach a judgment so early.

In guidelines released this summer, the FDA has said it will require a vaccine be at least 50% effective in preventing COVID-19 before it grants an approval. On safety, the agency wants to see data from thousands of study participants followed for at least six months after vaccination.

The FDA could also first grant an emergency use authorization, or EUA, which typically requires less data. Given the potential for widespread use of any cleared vaccine, however, FDA officials have indicated any emergency authorization would be more like an “EUA plus,” with a correspondingly higher bar.

Guidelines on the EUA process for coronavirus vaccines were supposed to be made public, but the document has become political because of how it could affect the chances of a vaccine being cleared for use before the Nov. 3 election.

While Pfizer and Moderna are likely to be the first with data, AstraZeneca and Johnson & Johnson could in theory announce results before the end of the year, too. AstraZeneca’s U.S. study is currently on hold due to safety concerns, while J&J just began its late-stage test last week.

Regeneron’s EB3 for Ebola virus infection

Regeneron is a leader in the race to develop antibody-based drugs for the new coronavirus. Its speedy progress is a product of the roadmap it developed battling another viral threat, Ebola, that could result in a drug approval next month.

Responding to the 2014 Ebola outbreak in West Africa, Regeneron deployed its antibody drug technology – which it’s used to make approved medicines for cancer and eye diseases, among others – against a viral infection.

Within months, Regeneron came up with REGN-EB3, a cocktail of three antibodies. But the 2014 outbreak passed, meaning Regeneron had to wait for the virus to return before it could prove the treatment’s worth.

Success finally came last year, when REGN-EB3 recorded positive results in a multi-drug trial amid a new flareup in the Democratic Republic of the Congo. The trial was stopped early after the antibody bested a drug called Zmapp at keeping patients alive after 28 days. Regeneron’s drug could be approved on Oct. 25, potentially becoming one of just a few available treatments for the lethal virus.

Originally posted on