Victoria Johnson joined the MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and now HCPLive. You can reach her at vjohnson@mjhlifesciences.com

Victoria Johnson

Welcome back to our recap of this month's news and updates from the 

March 2025 was a landmark month for FDA approvals, with game-changing therapies and medical innovations hitting the market. From the first new stroke treatment in decades to biosimilars expanding patient access, the agency delivered approvals that will shape healthcare for years to come. 

Breakthroughs in Crohn’s disease, ATTR-CM, and C3 glomerulopathy introduced new standards of care, while AI-powered diagnostics and novel drug formulations pushed the boundaries of precision medicine. Not every decision was an approval—delays and regulatory challenges also made headlines. Here’s a look at the most significant FDA developments from March 2025.

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 Approves Tenecteplase (TNKase) for Acute Ischemic Stroke

On March 3, 2025, Genentech announced that the FDA approved tenecteplase (TNKase) for the treatment of acute ischemic stroke (AIS), marking the first new stroke therapy approval in nearly 30 years. Tenecteplase, a tissue plasminogen activator, is administered as a single 5-second intravenous bolus, offering a faster and simpler alternative to alteplase. The approval was based on the phase 3 AcT trial, which demonstrated noninferiority to alteplase in efficacy and safety. 

FDA Approves Celltrion’s Denosumab Biosimilars for Bone Loss Indications

On March 3, 2025, Celltrion announced that the FDA approved denosumab-bmwo under the names Stoboclo and Osenbelt, a biosimilar to denosumab, for all approved reference indications, including osteoporosis and cancer-related bone loss. The approval was supported by phase 3 trial data demonstrating comparable efficacy, safety, and immunogenicity to the reference product. The biosimilars are expected to launch in the United States in June 2025.

FDA Approves First Generic of Rivaroxaban (Xarelto) for CAD, PAD


On March 4, 2025, the FDA approved the first generic formulation of rivaroxaban (Xarelto) 2.5 mg tablets, developed by Taro Pharmaceuticals, for reducing cardiovascular risk in adults with coronary artery disease (CAD) and thrombotic risk in adults with peripheral artery disease (PAD). The approval was supported by data from the COMPASS and VOYAGER-PAD trials, which demonstrated the efficacy of low-dose rivaroxaban combined with aspirin in lowering major cardiovascular and thrombotic events. The FDA emphasized that expanding access to safe and effective generics remains a priority to improve patient care.

FDA Approves 1 mg neffy Nasal Spray for Pediatric Anaphylaxis


On March 5, 2025, ARS Pharmaceuticals announced FDA approval of epinephrine nasal spray 1 mg (neffy) for treating type 1 allergic reactions, including anaphylaxis, in patients aged 4 years and older weighing 15 to less than 30 kg. The approval follows the August 2024 authorization of the 2 mg formulation for older children and adults. Supporting data included pharmacokinetic and pharmacodynamic studies demonstrating efficacy comparable to injectable epinephrine.

FDA Approves Revakinagene Taroretcel (ENCELTO) for MacTel

On March 6, 2025, Neurotech Pharmaceuticals announced FDA approval of revakinagene taroretcel-lwey (ENCELTO), the first and only treatment for Macular Telangiectasia type 2 (MacTel), a neurodegenerative retinal disease that causes progressive vision loss. Utilizing encapsulated cell therapy (ECT) technology, the implant delivers continuous therapeutic doses of ciliary neurotrophic factor (CNTF) to slow disease progression. Approval was based on positive phase 3 trial results demonstrating significant preservation of macular photoreceptors over 24 months, with availability expected in the U.S. by June 2025.

FDA Approves Furosemide Injection (Furoscix) for Edema in CKD

On March 6, 2025, scPharmaceuticals announced FDA approval of its supplemental New Drug Application (sNDA) for furosemide (Furoscix), expanding its indication to include the treatment of edema in patients with chronic kidney disease (CKD). The approval allows subcutaneous furosemide injection (80 mg/10 mL) to be used for fluid overload management in adults with chronic heart failure or CKD, including nephrotic syndrome. Expected to launch in April 2025, this expansion builds on prior approvals and supports at-home IV-equivalent diuresis, with ongoing development of an autoinjector for low-volume administration.

FDA Approves First Interchangeable Biosimilar for Omalizumab

On March 9, 2025, Celltrion announced FDA approval of omalizumab-igec (OMLYCLO) as the first interchangeable biosimilar to omalizumab (Xolair) and the first respiratory biosimilar for multiple allergic and respiratory conditions, including asthma and chronic spontaneous urticaria. Approval was based on a phase 3 trial demonstrating bioequivalence in chronic spontaneous urticaria, with similar efficacy, safety, and immunogenicity to Xolair. The designation may expand access and lower costs, particularly for patients with IgE-mediated food allergies, following omalizumab’s 2024 approval for reducing allergic reactions after accidental food exposure.

FDA Clears AI-Assisted CaRi-Plaque Image Analysis for CAD

On March 11, 2025, Caristo Diagnostics announced FDA 510(k) clearance for its AI-assisted CaRi-Plaque image analysis application, designed to assess coronary plaque and luminal stenosis from routine CCTA scans. The technology enables early detection of coronary artery disease by identifying plaque buildup and inflammation, potentially guiding earlier intervention and treatment. This approval expands the company's AI-powered cardiovascular imaging portfolio, following its CaRi-Heart platform, which has demonstrated predictive value for major adverse cardiovascular events.

FDA Approves Guselkumab (Tremfya) For Crohn Disease


On March 20, 2025, Johnson & Johnson announced FDA approval of guselkumab (Tremfya) for moderately to severely active Crohn’s disease, marking its fourth indication. The approval was supported by data from multiple Phase 3 trials, including GRAVITI and GALAXI studies, which demonstrated superiority over ustekinumab (Stelara) in achieving clinical remission and endoscopic response. Guselkumab, the first IL-23 inhibitor with both subcutaneous and intravenous induction options, offers a flexible treatment approach for patients with Crohn disease.

FDA Approves Oral Iptacopan (Fabhalta) as First C3 Glomerulopathy Therapy


On March 20, 2025, Novartis announced FDA approval of oral iptacopan (Fabhalta) for reducing proteinuria in adults with C3 glomerulopathy (C3G), marking the first approved treatment for this ultra-rare kidney disease. The approval was based on the Phase 3 APPEAR-C3G trial, which demonstrated a 35.1% reduction in 24-hour urine protein-to-creatinine ratio at six months, with sustained benefit at 12 months. This marks the second rare kidney disease indication for iptacopan, following its 2024 approval for primary IgA nephropathy.

FDA Approves Vutrisiran (AMVUTTRA) for ATTR-CM

On March 20, 2025, Alnylam announced FDA approval of vutrisiran (AMVUTTRA) for the treatment of adults with cardiomyopathy due to wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM). This makes vutrisiran the first approved RNAi therapeutic targeting cardiovascular mortality, hospitalizations, and heart failure visits in ATTR-CM. Approval is based on the phase 3 HELIOS-B trial, which showed a 28% reduction in all-cause mortality and cardiovascular events.

FDA Approves Gepotidacin (Blujepa) for Uncomplicated UTI

On March 25, 2025, the US FDA approved gepotidacin (Blujepa) for the treatment of uncomplicated urinary tract infections (uUTIs) in female adults and adolescents. This approval follows positive results from the Phase 3 EAGLE-2 and EAGLE-3 trials, where gepotidacin demonstrated non-inferiority or superiority to nitrofurantoin, the current standard of care. Gepotidacin, a first-in-class antibiotic, works by inhibiting bacterial DNA replication and is effective against most uropathogens, including drug-resistant strains.

FDA Issues CRL for Etripamil Nasal Spray (Cardamyst) in PSVT


On March 28, 2025, the US FDA issued a Complete Response Letter (CRL) for Milestone Pharmaceuticals' etripamil nasal spray (Cardamyst) for the treatment of paroxysmal supraventricular tachycardia (PSVT). The CRL did not raise concerns regarding the drug's clinical safety or efficacy but highlighted two key Chemistry, Manufacturing, and Controls issues, including the need for additional information on nitrosamine impurities and a facility inspection. Milestone plans to request a Type A meeting to address these issues and move forward with a resubmission.

FDA Approves Diazoxide Choline XR for Hyperphagia in Prader-Willi Syndrome


On March 26, 2025, the US FDA approved diazoxide choline extended-release tablets (Vykat XR) for the treatment of hyperphagia in adults and children aged 4 years and older with Prader-Willi syndrome (PWS). The approval, based on data from the phase 3 C602-RWP trial, marks the first FDA-approved therapy to address hyperphagia in PWS, demonstrating significant improvement in hyperphagia-related behaviors compared to placebo (P = .0022).

Fitusiran Approved for People With Hemophilia A or B, With and Without Inhibitors


On March 28, 2025, the US FDA approved fitusiran (Qfitlia) for routine prophylaxis to reduce bleeding episodes in patients aged 12 years and older with hemophilia A or B, with or without inhibitors. The approval was based on data from the phase 3 ATLAS program, where fitusiran prophylaxis demonstrated a 70% reduction in annualized bleeding rate compared to bypassing agent prophylaxis (

 = .0002). Sanofi’s therapy offers a less frequent dosing regimen, with some patients requiring as few as six doses per year.

Adding Treatment Options to the Hemophilia Landscape With Fitusiran’s Approval, with Margaret Ragni, MD, MPH

Ragni discussed unique advantages fitusiran offers over other approved therapies for hemophilia A or B.

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March 2025 saw significant FDA activity, with 12 approvals and one Refusal to File letter. Notable approvals include tenecteplase for acute ischemic stroke, marking a major advancement in stroke therapy, and the first generic rivaroxaban for CAD and PAD, enhancing access to cardiovascular care. Other key approvals include biosimilars for bone loss, a nasal spray for pediatric anaphylaxis, and treatments for rare diseases like MacTel and C3 glomerulopathy. The FDA also cleared AI-assisted imaging for CAD and approved new therapies for Crohn's disease, ATTR-CM, and uncomplicated UTIs.

This FDA News Month in Review provides a round-up of regulatory decisions from March 2025.


FDA News Recap: March 2025

Last Friday, the FDA approved fitusiran, under the name Qfitlia, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors.

The FDA approved fitusiran’s biologics license application off data from the phase 3 ATLAS program. Recent data from the ATLAS-OLE trial (NCT03754790) were presented during December’s 

American Society of Hematology (ASH) Annual Meeting and Exposition

 and demonstrated that fitusiran prophylaxis, in combination with appropriate bleed management guidelines, allowed fewer infusions and lower doses of clotting factor concentrates (CFC) or bypassing agents (BPAs) to treat breakthrough bleeding in people with hemophilia A or B.

spoke with Margaret Ragni, MD, MPH, an investigator on the ATLAS program and Professor of Medicine and Clinical and Translational Research, Division Hematology/Oncology, University of Pittsburgh, and Director, Hemophilia Center of Western PA, to learn more about fitusiran and how the approval improves the treatment landscape for both hemophilia A and B.

The fitusiran regimen yielded a 70% reduction in mean annualized bleeding rate (ABR) compared with BPA prophylaxis (

 = .0002) and was comparable with CFC prophylaxis (

 = .61). People receiving the fitusiran regimen also had lower annualized mean weight-adjusted consumption of CFCs and BPAs.

“What it really does is it makes your life a little easier, because each time you have to treat it interrupts what you're doing in everyday life, and, especially if it's intravenous, that's a problem. So, [fitusiran is] a simplification of management of this disease, and you know, it's not for everybody. So, it's all about talking with your patient, understanding what their preferences are, and understanding whether they would tolerate this type of drug,” Ragni told 

Ragni's disclosures include Sanofi, Pfizer, Alnylam, CSL Behring, Novo Nordisk, and others.

FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors. News release. FDA. March 28, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors

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 is potentially associated with metabolic disorders, new findings suggest, and resistin may provide a biomarker for disease severity, although the relationship may need additional research.

Magdalena Szczegielniak, from the department of dermatology at the Medical University of Lodz in Poland, worked alongside a team of other investigators in the analysis that led to these conclusions. The team set out to assess the inflammatory potential and association of psoriasis with metabolic and cardiovascular risk, looking at serum concentrations and correlations with related biomarkers and disease severity.

Szczegielniak and colleagues noted that an increase in markers can suggest inflammatory processes among patients that point to the risk of conditions accompanying psoriatic disease. They looked at serum concentrations of such hormones as resistin, a peptide hormone which is found both in adipose and non-adipose tissues and known to amplify systemic inflammation.

“The purpose of this study was to assess the inflammatory potential of psoriasis and the risk of metabolic and cardiovascular diseases in patients with psoriasis by analyzing the serum concentrations of homocysteine, adiponectin, resistin, leptin, and pentraxin 3 in pediatric patients with psoriasis and in a control group,” Szczegielniak and colleagues wrote.

 “Moreover, determination of the relationship between the studied markers and the severity of psoriatic lesions was carried out.”

The investigative team recruited patients from 3 pediatric dermatology centers between March 2020 - March 2022 for their analysis, all of whom had plaque psoriasis and were treated exclusively with topical drugs. The team evaluated a total of 103 participants, with 75 children involved in the study (47 girls and 28 boys) between the ages of 2 - 17 years.

The study’s control arm was made up of 28 children, with 15 girls and 13 boys, all of whom were without psoriasis. These individuals had been selected from patients presenting with non-inflammatory dermatologic conditions at the aforementioned dermatology centers.

The investigators looked at disease severity through an evaluation of Psoriasis Area and Severity Index (PASI) and of patients’ body surface areas (BSA). They conducted measurements of body weight and height to determine subjects’ body mass index (BMI). The team used BMI and patients’ ages to place them into categories labeled normal weight (<85th and >5th percentile), overweight (≥85th and <95th percentile), or obese (≥95th percentile).

The analysis required the collection among all subjects of fasting venous blood samples, 8 mL drawn into serum tubes and 4 mL into EDTA tubes. After centrifugation at 1000× g for 15 minutes, the resulting serum and plasma were aliquoted by the investigators into Eppendorf tubes and stored. Prior to their assessment, they thawed and homegenized the samples.

The investigative team used enzyme-linked immunosorbent assay (ELISA) kits to assess trial subjects’ serum concentrations of adiponectin, homocysteine, resistin, pentraxin 3, and leptin. The concentration of homocysteine was reported in µmol/L, pentraxin 3 in pg/mL, resistin and leptin in ng/mL, and adiponectin in µg/mL.

The team looked at correlations between biomarker levels and different variables such as BSA, PASI, and disease duration. They further assessed relationships that were shown to be statistically significant (

 < .05) using effect size measures such as Spearman’s rho. All of the statistical tests were 2-tailed, with the team setting statistical significance at 

 < .05, and effect sizes being reported where applicable.

Overall, the investigators concluded that children with psoriasis were shown to have higher serum levels of resistin, homocysteine, leptin, and pentraxin 3. They were also found to have lower serum levels of adiponectin as opposed to those included in the study’s control arm.

Notably, the investigative team highlighted a positive correlation that had been observed between resistin serum concentration and the severity of subjects’ psoriasis. Additionally, they reported that elevated resistin levels showed a link with higher scores on PASI and BSA.

“In our study, the levels of the analyzed biomarkers in psoriasis patients were not related to body weight, which implies that psoriasis without accompanying obesity may be an independent risk factor for systemic inflammation,” they wrote.1 “Resistin, in particular, appeared to be a potential marker of psoriasis severity, which could be used for monitoring disease progression. However, this relationship requires further investigation.”

Szczegielniak M, Lesiak A, Reich A, Opalińska A, Zakrzewski B, Arasiewicz H, Grabowski K, Nolberczak D, Narbutt J. Inflammation-Related Markers in Pediatric Psoriasis: Resistin as a Potential Marker of Psoriasis Severity. J Clin Med. 2025 Mar 3;14(5):1689. doi: 10.3390/jcm14051689. PMID: 40095687; PMCID: PMC11900389.

Johnston, A.; Arnadottir, S.; Gudjonsson, J.E.; Aphale, A.; Sigmarsdottir, A.A.; Gunnarsson, S.I.; Steinsson, J.T.; Elder, J.T.; Valdimarsson, H. Obesity in psoriasis: Leptin and resistin as mediators of cutaneous inflammation. Br. J. Dermatol. 2008, 159, 342–350.

Psoriasis may be linked to metabolic disorders, with resistin potentially serving as a biomarker for disease severity. A study by Magdalena Szczegielniak and colleagues analyzed serum concentrations of various biomarkers in pediatric psoriasis patients, revealing higher levels of resistin, homocysteine, leptin, and pentraxin 3, and lower adiponectin compared to controls. Resistin levels correlated with psoriasis severity, independent of obesity, suggesting its role as a marker for systemic inflammation and disease progression. Further research is needed to confirm these findings and explore resistin's potential as a clinical tool.

In this analysis, links between psoriasis and metabolic and cardiovascular risk were evaluated along with correlations between biomarkers and severity.

Study Indicates Resistin May Function as Biomarker for Psoriasis Severity


Welcome to HCPLive's 5 Stories in Under 5—your quick, must-know recap of the top 5 healthcare stories from the past week, all in under 5 minutes. Stay informed, stay ahead, and let’s dive into the latest updates impacting clinicians and healthcare providers like you!

Interested in a more traditional, text rundown? 

Top 5 Healthcare Headlines for March 23-30, 2025:

Zetomipzomib Shows Promise for Autoimmune Hepatitis in Phase 2a PORTOLA Trial

Zetomipzomib demonstrated steroid-sparing biochemical remissions in refractory autoimmune hepatitis patients, aligning with AASLD treatment guidelines in the Phase 2a PORTOLA trial.

Solriamfetol Significantly Reduces ADHD Symptoms in Axsome’s Phase 3 Trial

Solriamfetol met its primary endpoint in the Phase 3 FOCUS trial, significantly improving ADHD symptoms, with a higher proportion of patients achieving clinical response compared to placebo.

Sozinibercept for Wet AMD Misses Primary Endpoint in Phase 3 COAST Trial

Sozinibercept combined with aflibercept failed to meet the primary endpoint for visual acuity improvement in wet AMD, showing no added benefit over aflibercept monotherapy.

The FDA approved gepotidacin for uncomplicated urinary tract infections based on Phase 3 trials demonstrating non-inferiority to nitrofurantoin in female adults and adolescents.

FDA Issues CRL for Etripamil Nasal Spray (Cardamyst) in PSVT

The FDA issued a Complete Response Letter for etripamil nasal spray, citing manufacturing and control issues while confirming no concerns regarding its clinical safety or efficacy.

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An audio recap of the top 5 stories in healthcare news from the week of 03/23-03/2.