5 FDA decisions to watch in the fourth quarter of 2026

The coming approval decision for Akeso and Summit Therapeutics’ ivonescimab may be one of the biopharmaceutical sector’s most closely watched since the arrival of the ultra-successful cancer immunotherapies Keytruda and Opdivo in lung cancer a decade ago. 

Ivonescimab captured the attention of cancer researchers and drug executives in 2024, when it bested Keytruda in a study in non-small cell lung cancer in China. Those results suggested ivonescimab could upend lung cancer care in much the same way Keytruda did. They also sparked a flurry of investments in drugs that, like ivonescimab, target two proteins called PD-1 and VEGF that help drive cancer growth.

Ivonescimab’s potential has become the subject of intense debate since then. Investors and analysts have questioned the reliability of results from studies run only in China, and key global trials are still underway. Multiple competitors, among them Keytruda maker Merck & Co., are testing rival therapies in similar settings, too. 

Summit’s first U.S.submission also involves some controversy. The company is seeking use of ivonescimab in people whose lung cancer harbors an EGFR mutation and has progressed following treatment with a targeted therapy. But ivonescimab didn’t clearly extend lives upon a primary analysis in that trial. Only later did the results paint a rosier picture, suggesting it takes time for the drug’s “vaccinal effect to emerge,” wrote Stifel analyst Dara Azar, in July.

That situation has raised some questions as to whether the FDA might approve ivonescimab by the Nov. 14 decision deadline. The lack of a clear survival benefit, combined with a “minimal” contribution from patients outside of China on measures of tumor progression, makes for a pattern “the FDA has scrutinized closely in recent years,” wrote Leerink Partners’ Daina Graybosch. 

A filing leaning heavily on an “exploratory” survival analysis is “uncharted territory,” she wrote. — Jonathan Gardner

Roche investors have been closely tracking the progress of a breast cancer drug known as giredestrant. The Swiss company’s executive team has featured giredestrant heavily in quarterly presentations and, earlier this year, Roche Pharmaceuticals head Teresa Graham publicly declared it “could be our largest selling product” in the future.

Those words will be put to the test soon. By Nov. 30 and Dec. 18, respectively, a pair of FDA decisions are expected on giredestrant in two settings in the most common form of breast cancer. The first involves use in the “adjuvant” setting after surgery, while the second could lead to a clearance in metastatic disease following hormone therapy. 

Giredestrant is part of a class of oral “selective estrogen receptor degraders,” or SERDs. Multiple drug developers have touted oral SERDs as future blockbusters that could replace fulvestrant, an injectable therapy that has for decades been a treatment for certain breast cancers. Though four are now available, they haven’t yet proven in testing to be as broadly impactful as initially anticipated.

Roche, though, has seen giredestrant succeed in multiple treatment lines, including the post-surgical setting where other oral SERDs aren’t yet available. In that “adjuvant” study, a combination of giredestrant and hormone therapy delayed disease recurrence or death by 30% when compared to hormone therapy alone after surgery. Giredestrant has also, like some other oral SERDs, helped slow tumor progression when tested in more advanced disease.

Graham, on a July call with analysts, said that the “totality of the giredestrant data” from those two trials shows that Roche has “about 80% of the market covered.” The drug may be especially appealing to cancer patients who can’t take a commonly prescribed class of medications called CDK4/6 inhibitors, she said. 

“We do certainly think that there is quite a bit of opportunity for giredestrant,” Graham said.

Some analysts, like RBC Capital Markets’ Trung Huynh, are similarly optimistic. In a September note, Huynh called giredestrant “hugely underappreciated” and estimated it might rake in as much as $14 billion in peak annual sales. The bulk of those projected sales would come in the adjuvant setting, where treatment could have a “multi-year duration,” he wrote. 

Roche is “a commercial machine” in breast cancer given its existing products, Huynh wrote. That “should make adoption easier than consensus assumes.”

Still, Roche faced a setback earlier this year when giredestrant failed in the lucrative frontline setting. And others aren’t as convinced that the drug is destined for widespread use. 

“We are not saying giredestrant will not be a commercial drug, we just do not see the double digit billion sales potential the valuation implies,” Jefferies analyst Michael Leuchten wrote in July. — Gwendolyn Wu

Though Gilead Sciences is best known for its HIV drugs, the company has also been a leader in cancer cell therapy. Its $12 billion buyout of Kite Pharma nearly a decade ago handed the company two blood cancer treatments now sold as Yescarta and Tecartus. Those therapies are the foundation of a business that generated close to $2 billion last year. 

Yet that business is sputtering due to pressure from newer medicines. Gilead cited these “competitive headwinds” in its latest earnings report, which showed a 13% decline in first-half cell therapy sales compared to the same period a year prior. That slip followed a 7% slide in 2025 sales. 

A cell therapy Gilead purchased in one of the year’s biggest biopharma company acquisitions could help turn that business’ performance around. Called anito-cel, it was originally owned by the biotech Arcellx and is being developed for multiple myeloma. Testing showed a 96% response rate among recipients whose multiple myeloma had relapsed, or hadn’t responded to, at least three prior treatments. An FDA decision is expected by Dec. 23. 

Gilead has long been a believer in anito-cel’s potential, first partnering with Arcellx on the therapy in 2022 and then spending $7.8 billion in February to scoop up the company along with full anito-cel rights. But that therapy is set to enter a fiercely competitive space. Effective, marketed cell therapies from Johnson & Johnson and Bristol Myers Squibb are already available, as are a group of “bispecific” antibody therapies. Newer, potentially more convenient approaches are emerging too. 

Gilead executives have argued that anito-cel possesses certain safety advantages over existing multiple myeloma cell therapies, such as a lower risk of a side effect called parkinsonism. They’ve also contended that anito-cel could eventually become a “foundational treatment” across multiple treatment settings. 

At a recent investor conference, Chief Commercial Officer Johanna Mercier indicated that anito-cel could start fast once approved. Around 200 authorized treatment centers should be ready to administer anito-cel by the decision date, “basically 4 [times] more” than when one of Gilead’s competitors launched. “The intent,” she said, is “to really penetrate the fourth-line market quickly.” 

From there, the company’s plan is to “rapidly” move into earlier lines of care, including, eventually, newly diagnosed patients, CEO Daniel O’Day added.

“The profile of anito-cel is really highly differentiated,” said O’Day. “We think it’s best-in-disease.” 

They’ll have skepticism to overcome on Wall Street. Consensus projections have anito-cel sales topping out at $2.5 billion annually in 2035. Gilead has argued that estimate “considerably understates” the commercial opportunity. — Ben Fidler

Vertex Pharmaceuticals is used to having a near-total monopoly in cystic fibrosis treatment, where its arsenal of medicines prints more than $3 billion a quarter. Soon, though, the company plans to enter an increasingly competitive market for a different uncommon condition. And there, it will be playing catch-up.

The FDA should decide by the end of November whether to clear Vertex’s povetacicept as a treatment for IgA nephropathy, a chronic disease where an accumulation of antibodies progressively damages the kidneys. If greenlit, povetacicept would join a list of at least half a dozen approved therapies for this condition — which, according to some estimates, affects roughly 200,000 people in the U.S.

Supporting the drug are results from a late-stage clinical trial that were released in March. Thirty-six weeks into the study, researchers found that patients treated with Vertex’s drug as opposed to a placebo had a roughly 50% reduction in the amount of protein in their urine, a signal of better kidney health.

Wall Street analysts agreed the results were positive enough to warrant FDA approval, but were torn about how they stacked up to drugs like Otsuka’s Voyxact and Vera Pharmaceuticals’ fresh-to-market Trutakna. Otsuka’s medicine works by blocking an immune system protein known as APRIL, while Vertex’s and Vera’s inhibit both that protein and a second one called BAFF.

Following the povetacicept readout, Jefferies analysts claimed Vertex’s, Vera’s and Otsuka’s datasets looked “not clinically or statistically different for commercial uptake.” Meanwhile, Stifel analyst Paul Matteis wrote that key doctors in this space “have been divided on whether dual BAFF/APRIL inhibition is clearly best, and for now the answer still isn't clear.”

Even so, Matteis and others believe povetacicept has attributes that could still give it a leg up on rivals, like how it can be taken once a month at home through an injector. The drug’s safety, effectiveness and “favorable elements” should position it well to become a “key part of the treatment landscape even as a slightly later entrant, and to be successful across multiple other blockbuster indications,” analysts at RBC Capital Markets wrote in March.

The team at Raymond James has estimated that annual revenue from povetacicept could reach $1.5 billion by 2031. And at Cantor Fitzgerald, analysts expected peak yearly sales to hit $3 billion. — Jacob Bell

An experimental, RNA-based shot touted as a possible “functional cure” for a persistent viral liver infection could get to market in the U.S. as early as next month.  

Called bepirovirsen and developed by GSK and Ionis Pharmaceuticals for chronic hepatitis B cases, the drug is set to receive a decision from the Food and Drug Administration by Oct. 26. If approved as expected, bepirovirsen could provide a unique weapon against a lifelong infection the Centers for Disease Control and Prevention believes to affect 640,000 U.S.adults. 

Hepatitis B can be prevented by vaccination. When it isn’t, festering infections can spur major health problems, such as cirrhosis or liver cancer, later in life. 

Typically, patients with these infections take antivirals chronically to keep the virus at bay. Bepirovirsen represents a completely different alternative. Rather than only stopping hepatitis B from replicating, the drug also stimulates an immune response. That approach is supposed to more effectively clear out infected cells.

In two Phase 3 studies published in the New England Journal of Medicine, around a fifth of people who took bepirovirsen on top of antivirals saw their hepatitis B effectively wiped out, compared to none who got a placebo and antivirals. In a May note to clients, Leerink Partners analyst Mani Foroohar wrote that the results are likely to change the standard of care as the drug “sets a new bar for functional cure rates – a key focus of physicians and patients for finite-duration treatment.” 

GSK has a lot riding on bepirovirsen, which obtained its first approval, in Japan, in August. The drug is one of 15 coming launches its executives believe could potentially generate over $2 billion in peak annual sales. Foroohar believes the drug “has under-appreciated blockbuster potential in the long-term.”

A U.S. clearance would give Ionis, which has suffered multiple setbacks this year, a needed boost as well. Under its partnership with GSK, the company is eligible for tiered royalties of 10% to 12%. That could be a lucrative opportunity in a market William Blair analyst Myles Minter, in a May note, predicted to grow from $2.5 billion currently to over $7 billion by 2035. — Delilah Alvarado

Note: The FDA extended its review of deramiocel. Its new decision date is Nov. 22, 2026.

Over the last year or so, a cell therapy from Capricor Therapeutics has been swept up in the kind of regulatory turmoil the White House seems intent on bringing to halt. 

Capricor’s cell therapy is for the heart muscle weakness, or cardiomyopathy, that’s a leading cause of death in people with the rare disease Duchenne muscular dystrophy. Called deramiocel, it’s derived from a special type of heart cell known to help preserve function.

Capricor initially sought an FDA clearance largely on Phase 2 results showing the treatment helped improve measures of upper limb function as well as the heart’s ability to pump blood. It claimed to have aligned with agency officials in putting together deramiocel’s approval package, and was therefore taken by surprise when the FDA rejected its application last July.

That decision was one of many instances of drugmakers accusing the agency, under former leaders Marty Makary and Vinay Prasad, of backpedaling on previous agreements. Criticism of deramiocel’s rejection and others of rare disease medicines grew so loud that those cases were reportedly among the reasons the administration overhauled FDA leadership.

The agency has since revived the approval chances of one drug it had previously rejected, positioning Capricor’s therapy as perhaps the next high-profile test of FDA decisionmaking under the post-Makary regime.

Capricor has already accrued the data the FDA said it required turning back its therapy last year. The treatment hit two main goals in a study called Hope-3 that was much larger than the trial that had been at the center of its previous approval request.

The FDA is expected to make a decision by Aug. 22. Capricor could win a “priority review” voucher it could sell to other drugmakers should deramiocel get to market. — Ben Fidler

Note: The FDA approved oveporexton on Aug. 5, 2026. Read our coverage here.

Takeda could further cement its lead in what Wall Street expects to be a multibillion-dollar market if its drug oveporexton secures FDA approval over the next few months.

Oveporexton is part of an emerging class of medicines that stimulate “orexins” — proteins that regulate key brain functions like stress, energy, mood, learning and attention. Before the end of September, the FDA should decide whether positive data from a couple late-stage studies offer enough evidence to clear Takeda’s drug as a treatment for Type 1 narcolepsy.

That form of the sleep disorder is characterized not only by excessive daytime drowsiness, but also sudden bouts of muscle weakness known as cataplexy. In Takeda’s studies, oveporexton proved significantly better than a placebo at improving function, cognition and measures of wakefulness and muscle weakness in Type 1 narcolepsy patients.

Takeda has estimated that peak annual sales of its drug could reach $2 billion to $3 billion in this patient population alone. Additionally, because orexins have wide-ranging effects, research indicates that going after them is likely useful for treating conditions like ADHD as well other sleep disorders, from narcolepsy Type 2 to idiopathic hypersomnia.

Analysts therefore see big commercial opportunities for Takeda’s drug, along with candidates from Alkermes and Eli Lilly that are racing behind it. Indeed, a main argument among analysts is how valuable Takeda’s first-mover advantage will be. Alkermes, notably, kicked off this spring Phase 3 testing of its orexin-targeting agent alixorexton for both main types of narcolepsy. — Jacob Bell

Note: The FDA approved gedatolisib on July 14, 2026. Read our coverage here.

Cancer drugs targeting mutations to a gene called PIK3CA have attracted considerable interest from biotechs and investors in recent years. The latest one could reach the market soon, if U.S. regulators approve a drug from Celcuity by a July 17 deadline.

Celcuity’s stock price has rocketed nearly 700% since last year, when the company disclosed that its drug, gedatolisib, reduced the risk of disease progression or death in a late-stage study in people with a common form of breast cancer. That readout put Celcuity at the forefront of a new wave of therapies meant to improve upon PIK3CA-targeting breast cancer drugs like Novartis’ Piqray and AstraZeneca’s Truqap, which are associated with potentially harsh side effects.

Yet more recent results have made some investors skeptical of the drug’s prospects. Celcuity initially reported that gedatolisib was helpful for people without confirmed PIK3CA mutations. Additional testing in a different group of participants who have those mutations also unearthed a benefit, but a smaller one than had been observed in previous testing. The findings also raised questions among some Wall Street analysts as to how doctors might implement a “triplet” regimen involving hormone therapy and Pfizer’s Ibrance, or a “doublet” of just gedatolisib and hormone treatment. Celcuity has evaluated both regimens in its key trial, but the findings left doubts about which one is superior.

Still, some analysts and experts have rallied to Celcuity’s defense. Celcuity’s earlier-stage data were so striking they saddled the company with “lofty expectations” that were hard to meet, Leerink Partners’ Andrew Berens wrote in June. Study investigator Sara Hurvitz also noted in an interview that the drug has the chance to eventually “establish a new standard of care” for PIK3CA-mutated breast cancer.

The coming regulatory decision marks Celcuity’s first step towards that objective. An initial clearance would only cover people without PIK3CA mutations. Celcuity will later seek a broader approval via a supplementary application. — Delilah Alvarado

Note: The FDA approved mRNA-1010 on Aug. 5, 2026. Read our coverage here.

Moderna has endured more hardships than many of its biotech peers over the last few years. But an upcoming approval decision could provide a much-needed win, as well as indicate a friendlier stance from U.S. regulators going forward.

Moderna helped develop one of the world’s first COVID-19 vaccines, a landmark scientific achievement that helped it earn billions of dollars in revenue. But sales for that shot have declined significantly since the peak of the pandemic, and Moderna has struggled to find another big moneymaker. Meanwhile, the same messenger RNA technology it’s known for has become a political target in the U.S., leading Moderna to dial back infectious disease research and focus more on oncology.

An influenza vaccine the company has long been developing was swept up in that tumultuous journey. That vaccine, mRNA-1010, could become a unique weapon against seasonal influenza. Current shots are based on a slower production process that relies more on expert guesswork to determine which flu strains to target. Moderna’s technology enables shots to be designed and manufactured more quickly, which could lead to better matches and stronger efficacy.

Yet mRNA-1010’s development path has been highly unusual. Moderna initially brought it to regulators as part of a combination flu and COVID shot, but pulled that application following a request from the Food and Drug Administration for more data from the flu component. Moderna prepared a new application after gathering that information, only to see the FDA, under previous leadership, refuse to even review the submission. The agency reversed course after public backlash quickly mounted.

Since then, the FDA’s highest ranks have been overhauled and Moderna’s chances at a clearance have markedly improved. An FDA advisory panel recently voted unanimously that mRNA-1010’s benefits outweigh its risks in adults between the ages of 50 and 64 as well as the elderly. The meeting was a “big win” for Moderna, which views mRNA-1010 as important to revenue growth goals beginning in 2027, wrote William Blair analyst Myles Minter in a note to clients in June.

“While we hesitate to ascribe predictability to recent FDA decisions, we view the unanimous vote as a buildup of positive momentum for potential approval,” Minter added.

Moderna is seeking a traditional approval in people between the ages of 50 and 64, and an “accelerated” clearance in older adults. A decision will come by Aug. 5. — Delilah Alvarado

Note: The FDA approved enlicitide on July 16, 2026. Read our coverage here.

Injectable, cholesterol-lowering medicines known as PCSK9 inhibitors were once seen as the next big thing in heart medicine. Powerful study results, along with eye-popping price tags, led to multibillion-dollar sales projections as well as dire forecasts about the drugs’ likely effect on healthcare spending.

Those lofty expectations didn’t quite materialize because of a combination of insurer pushback, price cuts and nuanced clinical guidelines. Still, the top two medicines, Amgen’s Repatha and Regeneron Pharmaceuticals’ Praluent, have become blockbusters. Last year, Repatha generated sales of $3 billion and Praluent $1.4 billion.

Merck & Co. believes it might have better luck with a drug called enliclitide. A “macrocyclic” peptide taken via a daily pill, enliclitide could be far more convenient than medications that need to be injected every few weeks. Merck has also accumulated data suggesting that enlicitide can drop cholesterol levels as powerfully as its injectable counterparts, leading Leerink Partners analyst Daina Graybosch to claim in a recent research note that doctors “will view the options as largely interchangeable.”

That profile could shift the market for PCSK9 drugs toward enlicitide, particularly among primary care doctors who may be reluctant to prescribe injectable therapies. Merck executives have also argued that they can sidestep the reimbursement issues earlier PCSK9 medicines struggled with, as the company can manufacture enlicitide at a low cost and price it more competitively.

Merck hasn’t disclosed an FDA decision date for enlicitide. But it won a “national priority” voucher that could drastically speed up a review from the agency and, on a conference call in April, research chief Dean Li estimated that an approval could come in the second half of the year. Though the future of the voucher program is now unclear, in some cases, drugs that have received these fast-passes have been cleared within a couple months of a completed application.

Enlicitide’s approval is an important milestone for Merck, which views it as a key part of its plan to grow sales even after the patent expiration of its top-selling cancer immunotherapy Keytruda. — Jonathan Gardner