Many of the FDA’s decisions this quarter involve applications that have previously been delayed, declined or outright rejected, including one for an mRNA vaccine that became the center of controversy earlier this year.
The third quarter of 2026 is looking like a busy one for the FDA, with seven noteworthy decisions on its docket, including one that could mark a first in Alzheimer’s disease care and another that could justify a recent high-value buyout.
Read below for more.
Vera aims to challenge big names in IgAN arena
In one of Q3’s earliest decisions, Vera Therapeutics is looking to join the IgA nephropathy game with its investigational fusion protein atacicept. The drug is currently under FDA review with a target action date of July 7.
Backing atacicept’s application are data from the Phase 3 ORIGIN 3 study, in which the drug elicited a 46% decrease in proteinuria at 36 weeks versus baseline, according to a November 2025 press release. Atacicept also eliminated blood in the urine in 81% of patients.
These data paint a “compelling profile” for atacicept, Guggenheim Partners said in a Nov. 6 note to investors, strengthened even further by the drug’s “excellent safety profile that is comparable to placebo.”
If approved, atacicept would compete with Otsuka Pharmaceuticals’ APRIL inhibitor Voyxact, which won the FDA’s greenlight in November last year, and Novartis’ complement inhibitor Fabhalta. Vertex Pharmaceuticals, meanwhile, is close on Vera’s heels with its own fusion protein povetacicept, which has an FDA action date of Nov. 30.
Moderna’s mRNA flu shot nears D-day after sparking controversy
Moderna’s investigational flu vaccine made headlines in February when the FDA hit the company’s application with a refusal-to-file letter, declining even to give the submission the time of day. The agency at the time said that Moderna’s study used a comparator group that “does not reflect the best-available standard of care.”
A few days later, after strong blowback and a hastily arranged type A meeting with the biotech, the FDA reversed course and accepted an adjusted application for review. This time, Moderna is seeking full approval of its mRNA vaccine, dubbed mRNA-1010, in adults 50 to 64 years, and accelerated approval in people 65 and older. A decision is expected on or before Aug. 5.
Moderna is supporting the vaccine with many late-stage studies establishing its efficacy. One readout in June 2025 showed the mRNA shot was 26.6% more effective than a licensed comparator flu vaccine in adults 50 years and up.
Last month, the FDA’s Vaccines and Related Biological Products Advisory Committee unanimously recommended the mRNA flu shot’s full approval for adults aged 50 through 64, and accelerated approval for those 65 and up.
Capricor hopes for cell therapy comeback in Duchenne
By Aug. 22, the FDA is expected to release its decision on deramiocel, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy.
An approval here would serve as a vindication for deramiocel, which was initially rejected by the FDA last summer. At the time, the regulator said Capricor’s data package for the product failed to satisfy the “statutory requirement for substantial evidence of effectiveness.”
After deramiocel aced a Phase 3 trial, the FDA lifted its complete response letter in March, allowing the application to once again move forward. Capricor is seeking full approval of the candidate.
Deramiocel uses a rare subset of allogeneic cells drawn from the heart that can modulate the immune response and reduce fibrosis. The treatment works by helping maintain the function of cardiac and skeletal muscles. Data from the Phase 2 HOPE-2 study, which supported deramiocel’s initial application, demonstrated significant improvements in upper-limb performance and cardiac function over placebo.
Capricor’s resubmission now also includes data from the Phase 3 HOPE-3 study, which in December hit both its primary and secondary endpoints, showing statistically significant benefits in upper-limb function and a slowed decline in cardiac function as measured by left ventricular ejection fraction. Deramiocel also slowed disease progression and boosted overall functional performance, according to further data revealed in March.
Ultragenyx times two: Pair of gene therapies up for approval
Q3 is shaping up to be crucial for Ultragenyx, with two gene therapies lined up for FDA verdicts: DTX401 for glycogen storage disease type 1a (GSD1a), set for Aug. 23, and UX111 for Sanfilippo syndrome type A, due Sept. 19.
The upcoming decision date is especially pressing for UX111, which has already been rejected once. The FDA declined to approve the gene therapy in July 2025, citing manufacturing issues. Ultragenyx emphasized at the time that the denial was due to reasons “not directly related to the quality of the product.”
UX111 uses an adeno-associated virus (AAV) vector to deliver a functioning copy of the SGSH gene, which in patients with Sanfilippo syndrome type A is mutated, giving rise to a broad array of neurological and developmental symptoms. Around 1 in every 50,000 to 250,000 people globally have one of the four types of Sanfilippo syndrome, with A being the most common.
Meanwhile, GSD1a is caused by pathogenic mutations to the G6PC gene, produces an enzyme, G6Pase, that plays a role in the release of glucose from its stores. A deficiency in this activity can lead to severe hypoglycemia in between meals, as well as other complications that make the condition life-threatening. Patients with GSD1a are treated with cornstarch, which serves as an exogenous source of glucose.
DTX401 works by stabilizing the expression and activity of G6Pase. In Phase 3 development, the gene therapy led to a “significant and clinically meaningful” decrease in patients’ need for daily cornstarch intake, while also minimizing hypoglycemia episodes, according a company news release in February.
Biogen, Eisai ride out 3-month delay for starter dose of Leqembi Iqlik
After extending its review for 3 months, the FDA set a decision date of Aug. 24 for Eisai and Biogen’s application for the use of an under-the-skin formulation of their Alzheimer’s disease therapy Leqembi to initiate patients onto the treatment.
The FDA has already signed off on a subcutaneous version of Leqembi, now marketed as Leqembi Iqlik. However, the September 2025 approval only allows the use of the injection for maintenance treatment—that is, patients should first have completed 18 months of intravenous Leqembi.
A nod for Leqembi Iqlik in the induction setting would make it the first anti-amyloid therapy for at-home dosing throughout the entire treatment journey, the companies said in a January news release.
At the J.P. Morgan Healthcare Conference that same month, CEO Chris Viehbacher acknowledged that a nod is “going to be extremely important” and would help Leqembi compete with Eli Lilly’s Kisunla, which is infused monthly, versus Leqembi’s biweekly schedule. Kisunla’s current convenience edge “is going to go away once we have a subcutaneous formulation,” Viehbacher said.
Nuvalent nears NSCLC ruling for drug that attracted GSK buyout
Fresh off its $10.6 billion acquisition agreement with GSK, Nuvalent is nearing a potential approval for its ROS1 inhibitor zidesamtinib for the treatment of non-small cell lung cancer (NSCLC). The FDA’s verdict is expected on or before Sept. 18.
Nuvalent is backing its application with data from the Phase 1/2 ARROS-1 study. A readout in September 2025 showed that zidesamtinib elicited a 44% objective response rate in patients who had undergone prior treatment with a tyrosine kinase inhibitor. This figure improved to 51% when focusing on those who had only one previous line of treatment.
Zidesamtinib was one of the two stars of last month’s GSK takeover, alongside the ALK blocker neladalkib, also being proposed for NSCLC. The pharma believes both molecules have “multi-blockbuster potential,” according to GSK’s June 9 announcement. Truist Securities in a note the same day projected combined peak revenues for the two drugs to hit $3.5 billion.
Scholar Rock gives muscle atrophy drug another try
Capping off this list is Scholar Rock’s myostatin blocker apitegromab, which the biotech is proposing for the treatment of spinal muscular atrophy. The FDA’s target action date is September 30.
Apitegromab is a monoclonal antibody that inhibits the activation of myostatin, a protein that works to negatively regulate the growth of muscles. By blocking myostatin, apitegromab improves muscle mass and strength, as well as overall patient function and quality of life, according to Scholar Rock’s website.
Scholar Rock bore this out in the Phase 3 SAPPHIRE study, which in October 2024 showed significant motor improvements in patients treated with the antibody versus placebo.
In September 2025, however, the FDA handed Scholar Rock a rejection, flagging violations at a Novo Nordisk–owned manufacturing site where apitegromab was supposed to be produced. Other applications have also been tripped up by this particular plant, including Regeneron’s high-dose Eylea.
In March, Scholar Rock refiled its package for apitegromab, noting that while it will continue to engage the Novo facility, the biotech will also enlist a second fill-finish facility in the U.S. to “strengthen the apitegromab supply chain.”
