5 FDA decisions to watch in the third quarter of 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

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

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

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

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