The FDA is looking at a slew of label expansions this month, including one that could open up home-based treatments for Alzheimer’s disease.
With just a handful of target action dates lined up, May is shaping up to be a relatively quiet month for the FDA. Still, the regulator’s verdicts this month could have far-reaching implications for biopharma, including broadening patient pools for a couple of drugmakers and opening up at-home treatment for one of only two disease-modifying therapies on the market for Alzheimer’s disease.
Read below for more.
Argenx eyes ‘broadest label’ for myasthenia gravis drug
Argenx is looking to expand the label of its generalized myasthenia gravis (gMG) drug Vyvgart to cover patients who are seronegative for acetylcholine receptor (AChR) antibodies, a key disease marker. Approved in December 2021 for gMG, Vyvgart is currently only indicated for patients positive for AChR antibodies.
The FDA is reviewing this proposal, with a decision expected by May 10.
If approved, Vyvgart would boast “the broadest label” of all drugs in its class approved for gMG, analysts at William Blair wrote in an Aug. 25, 2025 note. Argenx would add approximately 11,000 more patients to its total addressable population, according to the firm.
Vyvgart is an IgG1 antibody fragment that works by blocking the neonatal Fc receptor (FcRn), in turn lowering the overall circulating levels of IgG, a type of immune system antibody that attacks AChR receptors and drives gMG pathology. Common gMG symptoms include muscle weakness that affects the limbs, leading to fatigue, difficulty walking and problems speaking or eating.
To support the proposal for a broader label, Argenx filed data from the Phase 3 ADAPT SERON study, which enrolled patients with three gMG subtypes, all of which were negative for AChR. Patients in the trial had gMG that was either MuSK-positive, LRP4-positive or negative for all three markers. Data showed “clinically meaningful” improvements in disease severity and activities of daily life after Vyvgart treatment, according to a January press announcement. Vyvgart was also well-tolerated, with no new safety concerns arising.
AstraZeneca, Daiichi Sankyo make neoadjuvant push for breast cancer drug
After notching a label expansion in January 2025, AstraZeneca and Daiichi Sankyo are keeping up the pace for their breast cancer antibody-drug conjugate (ADC) Enhertu. The pharma partners are now proposing to use the drug in a neoadjuvant setting—ahead of surgery—for patients with HER2-positive stage 2 or stage 3 breast cancer.
The FDA’s target action date for this application is May 18.
The companies are backing their bid with data from the Phase 3 DESTINY-Breast11 study. A readout in October 2025 showed that Enhertu—followed by a course of paclitaxel, trastuzumab and pertuzumab (THP)—resulted in a 67.3% pathologic complete response rate in patients with high-risk, locally-advanced disease who hadn’t yet undergone resection.
This effect was the “highest reported pathologic complete response rate seen in a Phase 3 registrational trial for HER2-positive early breast cancer,” AstraZeneca and Daiichi Sankyo claimed at the time.
Controls in DESTINY-Breast11 received a dose-dense cycle of doxorubicin and cyclophosphamide plus THP, yielding a pathologic complete response rate of 56.3%. The overall treatment effect was 11.2% in favor of the Enhertu regimen.
Enhertu has been on somewhat of a winning streak of late. In January 2025, the FDA signed off on the drug’s use in breast cancer patients with ultralow expression levels of HER2. Then, in December last year, the ADC again cleared the FDA’s regulatory bar for frontline use in patients with HER2-positive breast cancer—an approval that Jefferies analysts in a Dec. 16 note estimated would add $2 billion to $3 billion to peak sales.
Eisai, Biogen seek induction nod for SubQ Leqembi
In September 2025, Eisai and Biogen won the FDA’s go-ahead for an under-the-skin injection of their anti-amyloid Alzheimer’s therapy Leqembi—though the approval applied only to the maintenance use of the drug for patients who have already been on Leqembi for 18 months.
Now, the partners are proposing a similar subcutaneous injection formulation to initiate patients on the treatment. The FDA’s decision is expected on or before May 24.
Supporting the companies’ application are data from sub-studies of the Phase 3 Clarity AD trial, demonstrating that under-the-skin injections of Leqembi across various doses resulted in comparable drug exposure to an intravenous course given every two weeks. Biomarker and safety outcomes were likewise similar between the two regimens.
If approved, the subcutaneous formulation would allow for the at-home initiation of patients onto Leqembi therapy and open up a subcutaneous regimen for the entirety of their treatment journey, Biogen and Eisai said in a Jan. 25 release.
Cingulate’s ADHD drug nears FDA verdict
On or before May 31, the FDA is expected to release its decision regarding Cingulate Inc’s daily tablet CTx-1301 for attention deficit/hyperactivity disorder (ADHD).
CTx-1301 is a reformulated version of dexmethylphenidate, a stimulant of the central nervous system that has been validated for the treatment of ADHD and is the active ingredient in Novartis’ Focalin.
Cingulate’s version, which makes use of the company’s proprietary Precision Timed Release technology, is designed to deliver three doses at specific points during the day, according to the company’s website. This release profile is meant to “deliver the right amount of drug at the right time when patients need it,” ensuring rapid therapeutic activity and whole-day efficacy.
The FDA is reviewing CTx-1301 under the agency’s 505(b)(2) pathway, which allows sponsors to reference existing data on previously approved active ingredients, buffing it up with evidence of added benefit through a differentiated delivery mechanism, according to Cingulate. To satisfy this requirement, the biotech filed Phase 3 findings showing that CTx-1301 led to symptom relief assessed through different metrics, both in pediatric and adult populations.
Cingulate also showed that CTx-1301’s novel formulation was safe, with no serious treatment-emergent adverse events.
