As we turn the page on 2024 and look ahead to 2025, the medical community hopes to welcome another wave of transformative US Food and Drug Administration (FDA) decisions.

With several promising therapies in pipelines across a multitude of disease states, the beginning of the year promises to bring significant developments with the potential to redefine treatment paradigms across multiple therapeutic areas. From novel agents targeting rare conditions to innovative treatments for common diseases, the new year offers a host of exciting possibilities.

In this article, we highlight, in chronological order according to PDUFA date, 5 FDA decisions to watch in Q1 2025. These potential approvals are generating considerable anticipation and could have meaningful implications for both patients and healthcare providers.

5 FDA Decisions to Watch in Q1 2025

AXS-07

PDUFA: January 31, 2025
Indication: Acute Migraine
Summary: On September 04, 2024, Axsome Therapeutics announced that the FDA has accepted its resubmitted New Drug Application (NDA) for AXS-07, a novel treatment for acute migraine. AXS-07 combines meloxicam and rizatriptan, utilizing Axsome's MoSEIC technology to enable rapid absorption and longer plasma half-life. This submission follows a complete response letter from May 2022, which addressed manufacturing concerns but did not require additional clinical trials. The NDA is supported by positive data from the MOMENTUM and INTERCEPT Phase 3 trials, which demonstrated AXS-07's superiority over placebo in achieving pain freedom and reducing the most bothersome symptoms of migraine.

NT-501

PDUFA: March 18, 2025
Indication: Macular Telangiectasia type 2
Summary: On November 08, 2024, Neurotech Pharmaceuticals, Inc. announced that the FDA has extended the PDUFA goal date by 3 months for the BLA of NT-501 (revakinagene taroretcel) to treat Macular Telangiectasia type 2 (MacTel). The extension allows the FDA time to review additional data provided by the company in response to recent requests. NT-501 uses Neurotech's Encapsulated Cell Therapy platform to deliver CNTF, a neuroprotective protein, to slow retinal degeneration and improve visual outcomes in MacTel patients.

Vutrisiran

PDUFA: March 23, 2025
Indication: ATTR-CM
Summary: On November 25, 2024, Alnylam Pharmaceuticals announced that the FDA had accepted its sNDA for vutrisiran (Amvuttra), an investigational RNA interference therapeutic, for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM). At the time of submission, the FDA indicated that no advisory committee meeting would be held for review.
Vutrisiran, already approved for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN), works by significantly reducing both mutant and wild-type transthyretin (TTR). The sNDA is supported by positive results from the Phase 3 HELIOS-B trial, which demonstrated that vutrisiran reduced the relative risk of the primary composite endpoint (all-cause mortality and recurrent cardiovascular events) by 28% in the overall population (HR, 0.72; P = .01).

Gepotidacin

PDUFA: March 26, 2025
Indication: Uncomplicated Urinary Tract Infections
Summary: On October 16, 2024, GSK announced the FDA had accepted the NDA for gepotidacin, a first-in-class oral antibiotic for treating uncomplicated urinary tract infections (uUTIs) in female adults and adolescents.
Gepotidacin is a potential breakthrough treatment for uUTIs caused by drug-resistant bacteria, addressing an unmet need in a condition affecting over half of women. The NDA is supported by positive data from the pivotal EAGLE-2 and EAGLE-3 trials, where gepotidacin demonstrated non-inferiority and, in one trial, superior efficacy to nitrofurantoin, the current standard of care. If approved, gepotidacin would be the first in a new class of oral antibiotic treatment for uUTIs in over 20 years, according to GSK.

Etripamil Nasal Spray

PDUFA: March 27, 2025
Indication: PSVT
Summary: On March 28, 2024, Milestone Pharmaceuticals Inc. announced the resubmission of its NDA for etripamil, an investigational nasal spray for managing paroxysmal supraventricular tachycardia (PSVT). The resubmission follows guidance from a Type A meeting with the FDA after receiving a Refusal to File letter related to its initial March 2023 application.
The updated NDA includes restructured data sets and reformatting of certain data files to support the FDA's review, but no additional efficacy or safety data were required. On November 12, 2024, the company noted the FDA acceptance of their NDA in May 2024 and a March 2025 PDUFA date.