FDA News Month in Review: January 2025

Welcome back to our recap of this month's news and updates from the US Food and Drug Administration (FDA)!

In our January 2025 recap, we spotlight 18 FDA regulatory actions. Be sure to subscribe for a recap of the therapies, companies, and individuals moving the needle in healthcare during the past month, delivered on the first Saturday of each new month!

Want to hear from the experts? Look for our Related Content links underneath blurbs for insight into how this decision might influence practice moving forward.

FDA Grants Orphan Drug Designation to CNP-104 for Primary Biliary Cholangitis

On January 8, 2025, COUR Pharmaceuticals announced the FDA granted Orphan Drug Designation to CNP-104 for the treatment of primary biliary cholangitis (PBC). Based on positive topline data from a phase 2a trial presented at The Liver Meeting 2024, CNP-104 demonstrated favorable T-cell responses and a statistically significant reduction in liver stiffness. The designation highlights its potential as the first disease-modifying treatment for PBC.

FDA Accepts Revised Avacincaptad Pegol sNDA for Geographic Atrophy

On January 9, 2025, Astellas Pharma announced the FDA accepted the revised supplemental new drug application (sNDA) for avacincaptad pegol (IZERVAY) to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Based on a resubmission following a Complete Response Letter in November 2024, the FDA set a target action date of February 26, 2025. The sNDA aims to incorporate new two-year GATHER2 trial data into the US prescribing information.

Bayer Submits Finerenone Application to FDA for Treatment of Heart Failure

On January 10, 2025, Bayer submitted a supplemental new drug application (sNDA) to the FDA for finerenone (KERENDIA) for heart failure (HF) with LVEF ≥40%. Based on the Phase 3 FINEARTS-HF trial, finerenone demonstrated a 16% relative risk reduction in cardiovascular death and total HF events versus placebo. If approved, finerenone would be the first non-steroidal mineralocorticoid receptor antagonist (nsMRA) indicated for HF across the LVEF spectrum.

FDA Approves Mirikizumab (Omvoh) for Crohn’s Disease

On January 15, 2025, Lilly announced that the FDA approved mirikizumab (Omvoh) for the treatment of adults with moderately to severely active Crohn's disease (CD). Based on positive phase 3 VIVID-1 trial data, mirikizumab demonstrated superior clinical remission and endoscopic response rates compared to placebo at one year. This marks the first biologic in over 15 years to disclose two-year phase 3 efficacy data for CD at the time of approval.

Related Content: Marla Dubinsky, MD: Ushering In a New Era of Crohn’s Disease Treatment with Mirikizumab

FDA Approves Axatilimab 9 mg and 22 mg Vial Sizes for Chronic GVHD

On January 15, 2025, Incyte and Syndax Pharmaceuticals announced the FDA approved axatilimab-csfr (Niktimvo) in 9 mg and 22 mg vial sizes for chronic graft-versus-host disease (GVHD) after failure of ≥2 prior systemic therapies. Based on positive AGAVE-201 trial data, axatilimab is the first FDA-approved therapy targeting CSF-1R to address inflammation and fibrosis in GVHD. The newly approved vial sizes will be available for order in the US in early February.

FDA Accepts Plozasiran NDA for Familial Chylomicronemia Syndrome

On January 17, 2025, Arrowhead Pharmaceuticals announced the FDA accepted the New Drug Application (NDA) for investigational plozasiran to treat familial chylomicronemia syndrome (FCS), with a PDUFA target date of November 18, 2025. Based on positive Phase 3 PALISADE study results, plozasiran, an RNA interference therapeutic targeting APOC3, achieved an 80% median reduction in triglycerides and an 83% reduction in acute pancreatitis risk. The FDA previously granted Breakthrough Therapy, Orphan Drug, and Fast Track designations. Arrowhead plans further regulatory submissions in 2025.

FDA Approves Esketamine as First Monotherapy for Treatment-Resistant Depression

On January 21, 2025, Johnson & Johnson announced the FDA approval of esketamine (SPRAVATO) nasal spray as the first monotherapy for treatment-resistant depression (TRD). This decision follows a phase 4 placebo-controlled study demonstrating superior improvement in the Montgomery-Åsberg Depression Rating Scale (MADRS) at four weeks. Esketamine, an NMDA receptor antagonist, offers rapid antidepressant effects, with 22.5% achieving remission by day 28. Its safety profile remains consistent, with administration restricted to the SPRAVATO REMS Program due to risks of sedation, dissociation, and misuse.

Related Content: Gregory Mattingly, MD: What FDA’s Approval of Esketamine as Monotherapy Means for Treatment-Resistant Depression

Brelovitug Receives FDA Breakthrough Therapy Designation for Chronic Hepatitis Delta

On January 21, 2025, the FDA granted Breakthrough Therapy designation to Bluejay Therapeutics’ brelovitug (BJT-778) for chronic hepatitis delta (CHD), following phase 2 results showing 100% virologic response and up to 78% combined virologic response with ALT normalization. Brelovitug, a fully human IgG1 monoclonal antibody targeting HBsAg, aims to neutralize hepatitis B and D virions while potentially restoring antiviral immunity in CHB. Phase 2 data confirmed its strong antiviral activity and favorable safety profile across all dosing regimens, with long-term results expected in late 2025.

FDA Grants Tentative Approval to Generic Version of Rifaximin for IBS-D

On January 23, 2025, the FDA granted tentative approval to Amneal Pharmaceuticals’ rifaximin 550 mg tablets, referencing Bausch Health’s Xifaxan, for treating irritable bowel syndrome with diarrhea (IBS-D) in adults. Announced, the approval is pending litigation resolution. Rifaximin, a non-absorbable antimicrobial, targets gut bacteria with minimal systemic effects. The reference product, Xifaxan, was FDA-approved in 2015 based on Phase 3 trials demonstrating efficacy in symptom relief. The approval follows Amneal’s legal dispute with Bausch Health over its ANDA filing, which triggered a 30-month regulatory stay.

FDA Approves Label Update for Viloxazine Capsules to Include New Lactation Data

On January 27, 2025, Supernus Pharmaceuticals announced FDA approval of an updated label for viloxazine extended-release (Qelbree), incorporating new lactation data and pharmacodynamic insights. The update confirms a low likelihood of viloxazine transferring into breast milk and highlights its partial agonist activity at the serotonin 5-HT2C receptor and norepinephrine transporter inhibition. Originally approved for ADHD in children (2021) and adults (2022), viloxazine’s updated label aligns with FDA recommendations for lactation studies.

FDA Accepts New Drug Application for Bumetanide Nasal Spray

On January 27, 2025, Corstasis Therapeutics announced the FDA accepted its NDA for Bumetanide Nasal Spray, targeting a Prescription Drug User Fee Act (PDUFA) action date of September 14, 2025. The investigational nasal spray is designed as an outpatient therapy for fluid overload associated with congestive heart failure, liver, and kidney disease. Clinical data from the RSQ-777-02 trial showed that the nasal spray is absorbed effectively, with a 33% faster absorption rate than oral bumetanide, and offers similar efficacy and safety.

FDA Approves Semaglutide (Ozempic) for Type 2 Diabetes, Chronic Kidney Disease

On January 28, 2025, the FDA approved Novo Nordisk’s semaglutide (Ozempic) for the reduction of kidney disease progression, kidney failure, and death from cardiovascular disease in adults with type 2 diabetes and chronic kidney disease (CKD). Based on the phase 3b FLOW trial, semaglutide demonstrated a 24% relative risk reduction for kidney disease progression and cardiovascular-related death when semaglutide was added to standard care. The approval expands semaglutide’s indications, making it the most broadly indicated GLP-1 receptor agonist in its class.

Related Content: Diabetes Dialogue: Understanding Semaglutide (Ozempic) in CKD and T2D

FDA Grants Regenerative Medicine Advanced Therapy to Laru-Zova for XLRP

On January 28, 2025, Beacon Therapeutics announced FDA Regenerative Medicine Advanced Therapy (RMAT) designation for laruparetigene zovaparvovec (laru-zova) for X-linked retinitis pigmentosa (XLRP). Based on promising Phase 2 DAWN and SKYLINE trial data demonstrating improvements in low-luminance visual acuity, the designation facilitates accelerated development and regulatory interactions. Laru-zova, an investigational gene therapy targeting RPGR mutations, continues evaluation in the pivotal Phase 2/3 VISTA trial.

Ironwood Announces Rolling Apraglutide NDA Submission for Short Bowel Syndrome

On January 29, 2025, Ironwood Pharmaceuticals initiated a rolling New Drug Application (NDA) submission to the FDA for apraglutide for short bowel syndrome (SBS) requiring parenteral support. Data from the Phase 3 STARS trial demonstrated significant reductions in parenteral support volume and increased enteral autonomy in patients treated with apraglutide. Long-term extension data from STARS Extend will be included in the NDA. Ironwood is also restructuring, reducing its workforce by 50% to optimize resources ahead of a potential apraglutide launch.

FDA Grants 510(k) Clearance to First Lp(a) Blood Test in Molar Units

On January 29, 2025, the FDA granted 510(k) clearance to Roche’s Tina-quant® Lipoprotein (a) Gen.2 Molarity assay, the first blood test in the US to measure lipoprotein (a) [Lp(a)] in molar units. This assay is designed to assist in evaluating lipid metabolism disorders and assessing atherosclerotic cardiovascular disease (ASCVD) risk. The test provides clinicians with a more accurate assessment of cardiovascular risk by measuring the number of Lp(a) particles in the bloodstream.

FDA Clears IND for Frevecitinib Asthma Inhaler

On January 29, 2025, Kinaset Therapeutics announced FDA investigational new drug (IND) clearance for frevecitinib (KN-002) for asthma inadequately controlled by standard inhaled maintenance therapies. Based on positive Phase 1 results demonstrating clinically relevant reductions in fractional exhaled nitric oxide (FeNO) and a favorable pharmacokinetic profile, the company plans to initiate a Phase 2b dose-ranging trial in mid-2025.

FDA Approves Meloxicam and Rizatriptan (Symbravo) for Acute Migraine With or Without Aura

On January 30, 2025, Axsome Therapeutics announced FDA approval of meloxicam and rizatriptan (Symbravo) for acute migraine with or without aura. Based on positive data from the MOMENTUM and INTERCEPT Phase 3 trials, the combination therapy demonstrated superior efficacy over placebo in achieving pain freedom and symptom relief within two hours. The approval follows resolution of prior manufacturing concerns and introduces a novel treatment leveraging MoSEIC technology for rapid absorption and prolonged efficacy.

FDA Approves Suzetrigine, a Non-Opioid Option, for Treatment of Acute Pain

On January 30, 2025, the FDA announced the approval of suzetrigine (Journavx) for the treatment of moderate to severe acute pain in adults. Based on positive results from multiple Phase 2 and 3 trials, the selective NaV1.8 inhibitor demonstrated significant pain reduction compared to placebo in surgical pain studies. This approval marks the first new non-opioid pain class in over 20 years, offering a safer alternative to opioid-based treatments.