The second quarter is looking very busy for the FDA, which in April is expected to make a handful of very important decisions. One of them is for a rare disease with unmet needs, while the other, for the treatment of melanoma, can be a sign of how things can change in the institution after that. impending departure from the controversial leader.
Another key verdict in April is for Eli Lilly’s orforglipron, a weight loss pill that many expect will give Novo Nordisk oral Wegovy a run for its money.
here, BioSpace look at some of the biggest upcoming regulatory decisions expected in the next three months.
Biogen hopes to bounce back from High Dose Spinraza rejection
Following a surprised rejection in September of last year, Biogen is once again waiting for the FDA’s word on its high-dose formulation antisense oligonucleotide Spinraza, proposed for the treatment of spinal muscular atrophy (SMA). The agency is expected to release a decision on or before April 3.
Biogen supported its application with data from the Phase 2/3 DEVOTE study, which in September 2024 showed that a higher dose of Spinraza—including two loading doses of 50 mg every 14 days and a maintenance regimen of 28 mg every 4 months—led to better improvement in motor skills versus sham control. The higher-dose formulation of the drug also outperformed the standard-dose version in this regard, although the company at the time did not release its statistical analysis for this comparison.
At the recently concluded 2026 meeting of the Muscular Dystrophy Association, Biogen presented updated data from DEVOTE — as well as from an open-label Phase 3 study called ONWARD — confirming that the higher-dose Spinraza formulation was real better instead of sham in improving motor skills. Higher-dose Spinraza was also superior to sham in terms of secondary endpoints such as neurofilament light chain levels and event-free survival.
The FDA’s rejection of Spinraza in September 2025 was not because of the drug’s data package but otherwise. linked to manufacturing problems.
The Moment of Truth for Lilly’s Closely Watched Weight Loss Pills
In what can be the most consequential verdict for this year’s weight loss space, the FDA will make a decision on Eli Lilly’s oral obesity drug orforglipron on or before April 10.
Analysts expect orforglipron to help Lilly’s trizepatide franchise—made up of diabetes drug Mounjaro and weight management brand Zepbound—to exceed $ 100 billion in peak income. If approved, orforglipron will challenge Novo Nordisk’s oral Wegovy, which has a headstart three months after winning. approval at the end of last year.
Despite Novo’s first market advantage, Lilly may have an efficacy advantage. Data from Phase 3 reaches-3 A study released last month showed that orforglipron led to greater weight loss and better glycemic control than oral semaglutide—marketed as Rybelsus for diabetes—in patients with type 2 diabetes whose condition was not controlled by metformin.
FDA awarded orforglipron National Priority Voucher Commissioner in November 2025, promised a review period of 1-2 months. However, the agency late his decision in January by two weeks, according to Reuters.
If approved, Lilly would be ready to launch orforglipron quickly. Pharmacy has already stockpile approximately $1.5 billion of drugs, ready for distribution, and has increased production capacity since February 2024.
Target action dates for drugs sponsored by Sanofi, Boehringer Ingelheim and Disc Medicine have also been pushed back despite assurances of expedited reviews under the FDA’s new Commissioner’s National Priority Voucher program.
Replimune Gives Tumor Destroyers Another Try
Like Biogen, Replimune is looking to bounce back from rejection. The FDA is expected to release a decision for the company’s oncolytic immunotherapy RP1 for advanced melanoma by April 10.
regulators rejected RP1 in July last year, stated that the pivotal study of IGNYTE Replimune was insufficiently controlled and therefore could not support approval. This feedback, Replimine CEO Sushil Patel said at the time, “was not picked up by the agency during the mid- and end-of-cycle reviews.” Analysts at BMO Capital Markets had a clearer analysis of the rejection, suggesting that Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, played a role.
“Prasad has previously been critical of uncontrolled data approvals, and now we see that opinion underlined,” the firm wrote in a July 22, 2025 note to investors.
In October last year, Replimune resubmitted its data package for RP1, adding more data and additional analysis. It also has the potential to play in Replimune’s favor impending departure Prasad, who will leave the FDA at the end of April.
RP1 uses a proprietary and engineered herpes simplex virus that carries a fusion protein. Taken together, these constructs seek out and destroy cancer cells, while also altering the microenvironment and stimulating the body’s antitumor immune response. The RP1 is intended to be used in combination with Bristol Myers Squibb’s Opdivo.
BMO Capital Markets pointed to FDA leadership, and CBER Director Vinay Prasad in particular, as a potential factor in the agency’s decision to issue a full response letter to the Replimune RP1 viral treatment for advanced melanoma. The company’s stock tumbled 75% on Tuesday.
Axsome Awaits FDA Action on Alzheimer’s Agitation Drug
By April 30, the FDA will release verdict on Axsome Therapeutics’ oral drug AXS-05 for the treatment of agitation in patients with Alzheimer’s disease.
About 7 million people in the US have Alzheimer’s, according to Axsome, 76% of whom experience agitation characterized by distress, aggressiveness and sensitivity. AXS-05 combines two drugs—dextromethorphan and bupropion—and works by blocking NMDA receptors and activating sigma-1 receptors, while also inhibiting the CYP2D6 aminoketone enzyme.
In January 2025, Axsome was installed mixed late-stage data for medicine. In the Phase 3 ACCORD-2 study, AXS-05 reduced the risk of recurrence of agitation by 3.6-fold compared to placebo. A smaller Phase 3 study called ACCORD-1 also met its primary endpoint delaying the relapse time in 2022.
However, in the Phase 3 trial ADVANCE-2, which enrolled more than 400 patients, the drug Axsome failed to significantly outperform placebo in terms of eliminating agitation. Biotech also said at the time will advance with application.
AXS-05 has been approved in the US under the brand name Auvelity for the treatment of major depressive disorder.
After Win Maintenance, Biogen, Eisai Target SubQ expansion for Leqembi initiation
In September of last year, the FDA cleared the subcutaneous formulation of Biogen and Eisai’s anti-amyloid Alzheimer’s disease Leqembi, allows it to be delivered by injection under the skin instead of IV—but only in patients who have completed 18 months of treatment with the drug.
Now, the partners are looking to expand the subcutaneous injection, branded as Leqembi Iqlik, into the induction setting. FDA is currently reviewing the proposal and is expected to release a decision by May 24. In their data package, Biogen and Eisai provided data showing that weekly subcutaneous injections provide bioequivalent drug exposure to intravenous infusion given once every two weeks.
If approved, Leqembi Iqlik will be the first anti-amyloid therapy that patients can inject at home for induction and maintenance treatment. January 2026 press announcement from the company.
Approval “will also be very important,” Biogen CEO Christopher Viehbacher said said the investor at the company’s JP Morgan presentation in January. The chief executive noted that Biogen has competitors in the anti-amyloid antibody space, “and competitors offer once-monthly infusions versus our every-2-week infusions.” This competitor will be Eli Lilly’s Kisunla.
“That advantage will disappear once we have a subcutaneous formulation,” Viehbacher said.
Three years after the accelerated approval of its Alzheimer’s anti-amyloid therapy, Biogen-neck and neck in the market with Eli Lilly and its Kisunla offer-focused on the near-term FDA decision for the subcutaneous induction dose of Leqembi, presymptomatic reading in 2028 and the next generation candidate coupling.
Review Comes Close to AstraZeneca’s Lipid-Lowering Pills
AstraZeneca is looking to open a new drug class. The company is awaiting word from the FDA on its aldosterone synthase inhibitor baxdrostat for patients with uncontrolled or treatment-resistant hypertension, which, if approved, would be the first aldosterone synthase blocker to pass FDA muster, AstraZeneca. said in December last year.
The pharmacy has not set a target date for the application, only revealing that a decision should come in the second quarter.
Pharmaceuticals support baxdrostat with data from the Phase 3 BaxHTN study, where a dose of 2 mg of the drug showed a placebo-corrected decrease of up to 9.8 mmHg in systolic blood pressure, according to the August 2025 publication in The New England Journal of Medicine. BaxHTN used baxdrostat above the standard of care and also aced all of its secondary endpoints.
The drug, taken orally, works by inhibiting the enzyme aldosterone synthase, which is said to produce steroids that promote sodium retention, in turn maintaining blood pressure levels. AstraZeneca obtained baxdrostat from na $1.3 billion Takeover of CinCor Pharma in 2023.
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