Rilzabrutinib Preserves Platelet Response in ITP Through Extension Study, With David Kuter, MD

Rilzabrutinib has continued to demonstrate sustained platelet responses and symptom improvement in patients with 

, allowing patients to wean off concomitant corticosteroids, based on long-term extension data from the LUNA 3 trial.

67th American Society of Hematology (ASH) Annual Meeting and Exposition

 by David Kuter, MD, director of clinical hematology at Massachusetts General Hospital and professor of medicine at Harvard Medical School, these data reinforce the positive results from the base LUNA 3 trial, which saw rilzabrutinib improve physical fatigue and bleeding scores while maintaining a favorable safety profile.

“To be honest, I think that the major outcome of the long-term extension study is that patients were able to wean off or down on their concurrent ITP therapies,” Kuter told 

 in an exclusive interview. “Those concurrent therapies included things like corticosteroids and thrombopoietin receptors, and more than half of the patients declined or ceased those medications.”

LUNA 3 was a randomized, double-blind study conducted across 155 sites worldwide. Patients were eligible for enrollment if they had primary ITP with a duration of >6 months in patients aged 12 to <18 years and a duration of >3 months in patients aged ≥18 years. Additionally, patients were required to have an average of 2 platelet counts ≥5 days apart of <30,000/µL during the screening period and no single count >35,000/µL within 14 days prior to the first dose, as well as hemoglobin >9 g/dL within 1 week prior to study day 1.

Patients would be randomly assigned to receive either rilzabrutinib 400 mg or a matching placebo dose twice daily for 24 weeks. The primary outcome was durable platelet response during the last 6 weeks of the 24-week blinded treatment period. Secondary outcomes included the number of weeks with platelet count ≥50,000/µL or between ≥30,000/µL and <50,000/µL, the proportion of patients requiring rescue therapy, and the proportion of patients who achieved stable platelet response within a period of 24 weeks following initial achievement, among other endpoints.

After completing LUNA3, patients who had response (defined as platelet count ≥50x10^9/L or ≥30x10^9/L and at least doubled from baseline at ≥50% of visits without rescue therapy) during the last 8 weeks of the base trial. During the extension, patients would be given oral rilzabrutinib 400mg until loss of response on 2 consecutive visits or for safety reasons.

By April 1, 2025, a total of 169 patients had enrolled in the extension. Median age was 52 years (range, 18-80), and 72% were female. The median duration of ITP was 9.6 years (range, 0.3-42.7), and the median number of unique prior therapies was 4 (range, 1-9; 26% splenectomized). Additionally, 97% of patients had received corticosteroids. Median baseline platelet count was 18x10^9/L (range, 2-54x10^9/L).

At follow-up visits, median platelet counts ranged from 83-168x10^9/L through 33 months of the extension. During the first 12 months, patients had platelet counts ≥50x10^9/L or between 30-50x10^9/L, and at least doubled from baseline for an average of 88% visits (standard deviation [SD], 24%). Of patients who had received rilzabrutinib monotherapy (n = 22) or rilzabrutinib and concomitant corticosteroids (n = 47), median platelet counts at entry were 78x10^9/L (range, 20-371x10^9/L) and 115x10^9/L (range, 28-877x10^9/L), respectively, and ranged from 78-270x10^9/L and 90-160x10^9/L, respectively, through month 33.

Among the 34 patients in the extension who entered on concomitant corticosteroids, 10 discontinued, 2 reduced their dose ≥50% from the base study’s baseline, and 6 reduced their dose to <5 mg. Median platelet counts before and after corticosteroid discontinuation at last available visit were 148x10^9/L (range, 19-722x10^9/L) and 178x10^9/L (range, 3-405x10^9/L), respectively.

In addition to these results, Sanofi has also published data from the LUMINA series of trials investigating rilzabrutinib in warm autoimmune hemolytic anemia. The company plans to investigate this medication across a broader series of autoimmune conditions in the future.

“There are other autoimmune diseases for which ITP is a paradigm for therapy,” Kuter said. “I think that studying rilzabrutinib across the border in many autoimmune diseases is going to be a very important target as we move forward.”

Editors’ Note: David Kuter, MD, MSc, reports disclosures with Amgen, Bristol Myers Squibb, Daiichi Sankyo, Merck Sharp & Dohme, Medscape, Novartis, Pfizer, Principia, Regeneron, Sanofi, and others.

Kuter D, Zhang L, Ghanima W, et al. Reduction in corticosteroid use with rilzabrutinib and sustained response in adults with persistent/chronic immune thrombocytopenia in the long-term extension period of the Phase 3 LUNA3 study. Abstract presented at the 67th American Society of Hematology Annual Meeting. Orlando, FL. December 6-9, 2025.

Sanofi. Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA 3). ClinicalTrials.gov Identifier: NCT04562766. Updated February 10, 2025. Accessed December 12, 2025. 

https://www.clinicaltrials.gov/study/NCT04562766

Videos

Kuter discusses the efficacy and safety of rilzabrutinib in sustaining platelet responses and symptom improvement over the LUNA3 long-term extension.

News

news page, resources on the topics of disease- and specialty-specific medical news and expert insight can be found. Content includes articles, interviews, videos, podcasts, and breaking news on health care research, treatment, and drug development.

Clinical

clinical page is a resource for content on every specialty, disease, and topic available on our medical news site.

Hematology

Conference

 conference coverage page features articles, videos, and expert-led live coverage from major medical meetings throughout the year.

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for HCPLive. He is also the executive producer of two podcasts: Skin of Color Savvy and The Medical Sisterhood. Previously, he was the producer of the Lungcast podcast. He graduated from UC Berkeley and worked in multimedia journalism as a staff writer prior to joining MJH. You can contact him at tsmith@mjhlifesciences.com.

Dupilumab therapy shows the highest and most sustained 2-year drug survival among targeted 

 drugs, new data suggest, though Janus Kinase (JAK) inhibitors show a lower level of persistence largely due to ineffectiveness and adverse events.

These data resulted from a recent analysis of 2-year drug survival rates of JAK inhibitors as well as biologic therapies for patients living with atopic dermatitis. The study was written by investigators such as Lian F. van der Gang, a PhD candidate from University Medical Center Utrecht. 

“This study aimed to compare the 2-year drug survival of biologics and JAKi and identify associated predictors, and secondarily to evaluate the impact of new targeted therapies on dupilumab drug survival,” van der Gang et al wrote.

van der Gang and colleagues noted the importance of drug survival analysis as an indirect metric to indicate a treatment's effectiveness and tolerability. Additionally, they highlighted the metric's reflection on not only clinical outcomes but also decision-making among clinicians and patients. In addition to their aim of assessing JAKi and biologics in terms of drug survival, the investigators' secondary aim was to look at whether the introduction of additional targeted drugs impacted survival rates for dupilumab.

In this evaluated, adults aged 18 years or older with the skin disease were included who were also treated in routine clinical practice with abrocitinib, dupilumab, tralokinumab, upadacitinib, or baricitinib. The multi-center BioDay registry was used to find patients, and these individuals had initiated therapy in the period between January 2021 - October 2024. Drug survival was determined by van der Gang and coauthors via a Kaplan–Meier analyses.

It was stratified according to prior exposure to biologics or JAKi as well as by participants' rationale for drug discontinuation. Those who had remained on drug at the conclusion of follow-up, were lost to follow-up, or ended their treatments because of a desire to become pregnant or due to well-controlled disease were censored.

The investigative team used Cox proportional hazards models to assess any differences between medications and predictors of drug cessation. The team handled missing values via multiple imputation with 50 datasets. Necessary adjustments for multiple comparisons were conducted through the false discovery rate. For van der Gang et al's secondary analysis, all dupilumab treatment episodes between 2017 - 2024 were included, with the availability of alternative targeted drugs incorporated as a time-dependent variable.

Overall, the investigators assessed 227 tralokinumab, 865 dupilumab, 144 abrocitinib, 106 baricitinib, and 241 upadacitinib treatment episodes over the course of the study. Dupilumab was most often prescribed as the initial targeted medication, accounting for 82.1% of treatment episodes. Additionally, they found tralokinumab was implemented as first-line therapy in 44.9% of cases.

In contrast, van der Gang and colleagues found JAK inhibitors were predominantly initiated following prior targeted treatments, including abrocitinib in 125 treatment episodes (86.8%), baricitinib in 75 episodes (70.8%), and upadacitinib in 203 episodes (84.2%). Patients who were biologic- and JAKi-naïve saw drug survival rates at 1 and 2 years of 84.9% and 77.3% for dupilumab, 60.2% and 27.1% for abrocitinib, 72.5% and 47.6% for tralokinumab, 46.7% and 42.8% for baricitinib, and 72.6% and 63.5% for upadacitinib, respectively.

The investigative team found lower drug survival generally among subjects showing prior biologics or JAKi exposure. In this experienced cohort of patients, 1- and 2-year survival rates were 53.7% and 27.8% for abrocitinib, 82.9% and 68.1% for dupilumab, 29.6% and 22.4% for baricitinib, 61.3% and 49.4% for upadacitinib, and 59.8% and 49.6% for tralokinumab. Among both naïve and experienced individuals, the team concluded treatment discontinuation appeared most often due to insufficient drug effectiveness (20.6%), followed by adverse events 13.7%. 

During treatment discontinuations resulting from issues with efficacy, van der Gang and coauthors found 95.7% of such patients had an Eczema Area and Severity Index (EASI) score exceeding 7 and/or a Numeric Rating Scale (NRS) score for pruritus over 4. The remaining individuals discontinued treatment mainly due to persistent involvement of the face or hands or due to the initiation of concomitant systemic drugs.

In the study's secondary analysis, the investigators observed a link between the availability of additional targeted drugs and a marked reduction in dupilumab persistence. Specifically, they identified a twofold higher risk of therapy discontinuation following entrance of new options into the market (HR 2.0, 95% CI 1.4–2.5; 

“This study highlights that the absence of alternative targeted therapies results in significantly longer drug survival, indicating that treatment availability influences drug survival outcomes," they concluded.

 "Additionally, drug survival was generally lower in biologic-/JAKi-experienced patients."

LF van der Gang, CM Boesjes, NPA Zuithoff, et al. Drug Survival in Atopic Dermatitis: Comparison of Biologics and JAK Inhibitors in the BioDay Registry. Allergy (2025): 1–5, 

van den Reek JMPA, Kievit W, de Jong EMGJ, et al. Drug Survival Studies in Dermatology: Principles, Purposes, and Pitfalls. J Invest Dermatol. 2015 Jul;135(7):1-5. doi: 10.1038/jid.2015.171. PMID: 26066896.

Articles

Dupilumab demonstrates the highest and most sustained 2-year drug survival among targeted therapies for atopic dermatitis, outperforming Janus Kinase (JAK) inhibitors, which show lower persistence due to ineffectiveness and adverse events. The study analyzed drug survival rates using the BioDay registry, focusing on biologics and JAK inhibitors. Dupilumab was frequently the initial treatment, while JAK inhibitors were often used after other targeted therapies. Drug survival was lower in patients with prior biologic or JAK inhibitor exposure. The availability of new targeted therapies reduced dupilumab persistence.

Investigators compared the 2-year drug survival rates of biologics and JAKi in atopic dermatitis, in addition to evaluating associated predictors.

Atopic Dermatitis

Atopic Dermatitis condition center page, audiences can find a variety of coverage mediums for medical news and updates, including articles, video interviews, podcasts, and breaking news on dermatitis research, treatment, and drug development.

Dermatology

The HCPLive dermatology page is a resource for medical news and expert insights on skin disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for dermatologic conditions, and more.

Dupilumab Shows Greatest Rate of 2-Year Drug Survival Among Atopic Dermatitis Drugs

, associate clinical professor of medicine and pediatrics at the David Geffen School of Medicine, University of California, Los Angeles.

Rezpegaldesleukin Shows Promise for AD, Comorbid Asthma, With Jonathan Corren, MD

, a first-in-class IL-2 receptor pathway agonist, significantly improved skin outcomes and 

moderate-to-severe atopic dermatitis (AD)

, according to late-breaking data from the Phase 2b REZOLVE-AD trial presented at the 2025 American College of Allergy, Asthma, & Immunology (ACAAI) Annual Scientific Meeting in Orlando, Florida.

“This is a very new approach by trying to augment T regulatory activity, which has a potential for controlling inflammation in a lot of different ways, and trying to do it without any immunosuppression,” investigator Jonathan Corren, MD, associate clinical professor of medicine and pediatrics at the David Geffen School of Medicine, University of California, Los Angeles. “That's what really makes this a unique approach. What we saw in atopic dermatitis was extremely encouraging. [I am] looking forward to larger trials to hopefully confirm these same findings.”

Rezpegaldesleukin selectively expands regulatory T-cell activity, controlling inflammation without broad immunosuppression,” said Dr. Jonathan Corren, Associate Clinical Professor at UCLA. “In patients with both AD and asthma, we observed meaningful reductions in Asthma Control Questionnaire-5 scores, suggesting benefits beyond the skin.”

The REZOLVE-AD trial enrolled 393 adults, including 99 with a history of asthma. Participants received subcutaneous rezpegaldesleukin at 24 μg/kg every 2 weeks, 18 μg/kg every 2 weeks, 24 μg/kg every 4 weeks, or placebo over a 16-week induction period.

Patients in the 24 μg/kg every 2 weeks group achieved statistically significant improvements across all endpoints. At week 16, At week 16, rezpegaldesleukin produced significant improvements in Eczema Area and Severity Index (EASI; 

 <.05), validated Investigator Global Assessment (vIGA-AD; 

 <.05), and Numerical Rating Scale Itch (NRS-Itch; ≥4-point reduction, 

 <.01). Benefits were consistent regardless of baseline disease severity, sex, geographic region, or asthma comorbidity.

Patients with partially controlled or uncontrolled asthma showed the most pronounced reductions in ACQ-5 scores. Among those with uncontrolled asthma at baseline, 75% achieved clinically meaningful improvement.

Crossover data from 42 placebo patients who switched to high-dose rezpegaldesleukin at week 16 confirmed sustained efficacy at week 24. At crossover, 60% of patients achieved EASI-75, 37% reached EASI-90, 33% achieved vIGA-AD 0/1, and 50% reported ≥4-point reductions in Itch NRS. These results support a 24-week induction period before transitioning to maintenance therapy.

Safety was favorable, with no serious immunosuppressive, systemic, or ocular adverse events reported. The US Food & Drug Administration granted Fast Track designation for rezpegaldesleukin in patients aged ≥ 12 years with moderate-to-severe AD not adequately controlled with topical therapies, announced by Nektar Therapeutics on February 10, 2025.

“This research represents a very new way of looking at the modulation of allergic atopic diseases, from the perspective [of] you're not blocking a single mediator in the case of IL-5 or IL-13, IL-31, or IgE, but you're really trying to change the profile of regulatory cells that can dampen the entire inflammatory response all at once,” Corren said. “This gives us high hopes that, ultimately, maybe there will be better effects, longer-lived effects, which will be unique and differentiated from what we currently have available to us.”

Derman C. Rezpegaldesleukin Shows Strong Phase 2b Results for Atopic Dermatitis, Asthma. HCPLive. Published November 21, 2025. Accessed December 12, 2025. 

https://www.hcplive.com/view/rezpegaldesleukin-shows-strong-phase-2b-results-atopic-dermatitis-asthma

Nektar Therapeutics Receives Fast Track Designation for Rezpegaldesleukin for the Treatment of Moderate-to-Severe Atopic Dermatitis. Nektar Therapeutics. Published February 10, 2025. Accessed December 12, 2025. 

https://ir.nektar.com/news-releases/news-release-details/nektar-therapeutics-receives-fast-track-designation

Phase 2b REZOLVE-AD trial data show rezpegaldesleukin improves skin symptoms and asthma control in adults with moderate-to-severe atopic dermatitis.

Pulmonology

Infectious Disease condition center page is a comprehensive resource for clinical news and insights on respiratory health. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for asthma, COPD, interstitial lung disease, COVID-19, and more.

Asthma

The HCPLive asthma page is a comprehensive resource for clinical news and insights on asthmatic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for allergic and severe asthma, and more.

Welcome to The HCPFive, your go-to roundup for the latest healthcare news and breakthroughs, curated specifically for busy healthcare professionals.

Each week, we highlight 5 key developments or headlines from healthcare that you need to know—whether it's a cutting-edge treatment, regulatory updates, or innovations shaping the future of medicine. This week's top stories include the US Food and Drug Administration approvals of omidubicel-onlv (Omisirge) for the treatment of severe aplastic anemia, etuvetidigene autotemcel (Waskyra) for Wiskott-Aldrich syndrome, gepotidacin (Blujepa) for patients with uncomplicated urogenital gonorrhea, trofinetide (Daybue Stix) for oral solution for Rett syndrome, and berotralstat (Orladeyo) oral granules for patients with hereditary angioedema (HAE).

With The HCPFive, you'll get the essential takeaways to stay informed and ahead of the curve. Here’s your quick dive into the top stories for the week of December 7, 2025—let’s jump in!

FDA Approves Omidubicel-onlv (Omisirge) As First Cellular Severe Aplastic Anemia Therapy

On December 8, 2025, the FDA approved Ayrmid’s omidubicel-onlv (Omisirge) for the treatment of severe aplastic anemia, making it the first hematopoietic stem cell transplant therapy for this indication. The treatment addresses the limitations of umbilical cord blood as a source including delayed hematopoietic recovery and increased infections and provides additional graft options for patients with severe aplastic anemia who need hematopoietic stem cell transplant.

FDA Approves Etuvetidigene Autotemcel (Waskyra) For Wiskott-Aldrich Syndrome

On December 9, 2025, the FDA approved etuvetidigene autotemcel (Waskyra), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome. It is indicated for pediatric patients ≥ 6 months of age and adults with Wiskott-Aldrich syndrome who have a mutation in the 

 gene and for whom hematopoietic stem cell transplantation is appropriate and no suitable human leukocyte antigen-matched related stem cell donor is available.

FDA Approves Gepotidacin as Oral Treatment of Uncomplicated Urogential Gonorrhea

On December 11, 2025, the FDA approved GSK’s gepotidacin as an oral option for adult and pediatric patients ≥ 12 years of age weighing ≥ 45 kg who have limited or no alternative options for the treatment of uncomplicated urogenital gonorrhoea caused by susceptible strains of 

. The decision follows the FDA approval of gepotidacin earlier this year as an oral treatment for female adult and paediatric patients ≥ 12 years of age weighing ≥40 kg with uncomplicated urinary tract infection.

FDA Approves New Powder Formulation of Trofinetide (Daybue) for Rett Syndrome

On December 12, 2025, the FDA approved Acadia Pharmaceuticals’ trofinetide (Daybue Stix) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients ≥ 2 years of age. The new formulation is expected to deliver the same efficacy and safety profile of trofinetide oral solution, while offering children and adults living with Rett syndrome new flexibility and choice regarding the dose volume and taste of their trofinetide treatment.

FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE

On December 12, 2025, the FDA approved BioCryst’s berotralstat (Orladeyo) oral granules for patients with HAE aged 2 – 11 years. The decision expands upon berotralstat’s 2020 FDA approval for the prevention of HAE attacks in adults and children aged ≥ 12 years.

Brooks A. FDA Approves Omidubicel-onlv (Omisirge) As First Cellular Severe Aplastic Anemia Therapy. HCPLive. December 8, 2025. Accessed December 12, 2025. 

https://www.hcplive.com/view/fda-approves-omidubicel-onlv-omisirge-first-cellular-severe-aplastic-anemia-therapy

Brooks A. FDA Approves Etuvetidigene Autotemcel (Waskyra) For Wiskott-Aldrich Syndrome. HCPLive. December 9, 2025. Accessed December 12, 2025. 

https://www.hcplive.com/view/fda-approves-etuvetidigene-autotemcel-waskyra-wiskott-aldrich-syndrome

Smith T. FDA Approves Gepotidacin as Oral Treatment of Uncomplicated Urogential Gonorrhea. HCPLive. December 11, 2025. Accessed December 12, 2025. 

https://www.hcplive.com/view/fda-approves-gepotidacin-oral-treatment-uncomplicated-urogential-gonorrhea

Brooks A. FDA Approves New Powder Formulation of Trofinetide (Daybue) for Rett Syndrome. HCPLive. December 12, 2025. Accessed December 12, 2025. 

https://www.hcplive.com/view/fda-approves-new-powder-formulation-of-trofinetide-daybue-for-rett-syndrome

Derman C. FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE. HCPLive. December 12, 2025. Accessed December 12, 2025. 

https://www.hcplive.com/view/fda-approves-oral-berotralstat-children-aged-2-11-years-hae

Recent FDA approvals have introduced significant advancements in treatment options for various conditions. Omidubicel-onlv (Omisirge) is now the first cellular therapy for severe aplastic anemia, addressing limitations of umbilical cord blood. Etuvetidigene autotemcel (Waskyra) offers a gene therapy solution for Wiskott-Aldrich syndrome. Gepotidacin provides an oral treatment for uncomplicated urogenital gonorrhea. Trofinetide (Daybue Stix) introduces a new powder formulation for Rett syndrome, enhancing treatment flexibility. Berotralstat (Orladeyo) oral granules expand treatment options for hereditary angioedema in younger children.

Stay updated with the latest healthcare breakthroughs, including several new FDA approvals, in this week's essential news roundup.

Allergy

Allergy condition center page is a comprehensive resource for clinical news and insights on allergic and inflammatory diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for food and seasonal allergies, as well as allergic asthma and related conditions.

The HCPFive: Top News for Healthcare Providers from the Week of 12/07

Ryan Livingston joined the MJH Life Sciences team in March of 2025, shortly after graduating from TCNJ with a bachelor’s degree in English. He enjoys creative writing, reading, and scuba diving. He can be reached at rlivingston@mjhlifesciences.com.

Image Credit: US Food and Drug Administration

Announced by parent company Milestone Pharmaceuticals on December 12, 2025, the 

 has approved etripamil (Cardamyst) nasal spray for the conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults.

The decision marks the first time that more than 2 million people in the US with PSVT will have a rapid-acting treatment option they can self-administer outside the emergency department or other healthcare setting. As described in the release from Milestone, etripamil is expected to be available in retail pharmacies in the first quarter of 2026.

“Some people with PSVT have endured years of anxiety, fearing their next episode and the stress and disruption of emergency department visits,” James Ip, MD, an etripamil investigator, said in a statement.

 “CARDAMYST will give many of them the ability to administer a medication themselves that can quickly stop their PSVT episode and potentially avoid a hospital trip or a call to emergency services.”

Etripamil is a novel calcium channel blocker nasal spray developed to treat frequent and highly symptomatic episodes of 

paroxysmal supraventricular tachycardia (PSVT)

. It is designed as a rapid response therapy to be self-administered by patients, bypassing the need for medical oversight. It offers patients more control over their condition via active management.

Etripamil’s efficacy has been proven across various trials in the overarching NODE program, including the NODE-1, NODE-301 parts 1 and 2, NODE-302, and NODE-303 trials. A collective analysis of all of these trials was presented at the American Heart Association’s 2025 Scientific Sessions in November 2025, which highlighted the overall efficacy, safety, and test-dose tolerability of etripamil in patients with PSVT.

During this collective analysis, efficacy measures included the proportion of patients converting from PSVT to normal sinus rhythm (SR) within 30 minutes and the median times to conversion across all the included phase 3 studies. Safety data for treatment-emergent adverse events (TEAEs), serious adverse events, and test dose tolerability from phase 2 and 3 studies were also summarized.

The Kaplan-Meier estimate during this analysis displayed a substantial 59.6% of patients exposed to etripamil converted to normal SR within 30 minutes, with a median time to conversion of 18.5 minutes (95% CI, 15.7-21). The proportions of placebo group patients converting from SVT to SR by 30 minutes ranged from 26.7% to 34.7%, while estimates of conversion with etripamil at 60 minutes for the individual studies ranged from 63.2% to 73.5%.

Additionally, the combined analysis displayed largely mild and transient treatment-emergent adverse events, primarily nasal congestion, nasal discomfort, throat irritation, rhinorrhea, and epistaxis. A total of 1107 patients took ≥1 etripamil test dose while in SR, and no significant change in average baseline heart rate or blood pressure was observed within 45 minutes of administration. Test dose failure occurred in only 1.4% of patients (n = 16). Investigators determined no need for a pretreatment dose, highlighting etripamil’s favorable tolerability.

“CARDAMYST is a novel at-the-ready treatment option that addresses the unpredictable impact of PSVT by offering patients the freedom to manage episodes anytime and anywhere,” said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals.

 “The FDA approval of CARDAMYST is a watershed moment for Milestone and a gratifying event for our team members, patients, clinical investigators, and health care providers who participated in the development program, all of whom I sincerely thank for their dedication, counsel, and collaboration toward this important achievement.”

Milestone Pharmaceuticals. Milestone Receives FDA Approval of CARDAMYST™ (etripamil) as First and Only Self-Administered Nasal Spray for Adults with Paroxysmal Supraventricular Tachycardia (PSVT). December 12, 2025. Accessed December 12, 2025. https://www.globenewswire.com/news-release/2025/12/13/3204944/0/en/Milestone-Receives-FDA-Approval-of-CARDAMYST-etripamil-as-First-and-Only-Self-Administered-Nasal-Spray-for-Adults-with-Paroxysmal-Supraventricular-Tachycardia-PSVT.html

2. Gardner K. Milestone Pharmaceuticals Announces FDA Acceptance of the Company’s Response to the CRL for CARDAMYST (etripamil) Nasal Spray. Milestone Pharmaceuticals. July 11, 2025. Accessed December 9, 2025. 

https://investors.milestonepharma.com/news-releases/news-release-details/milestone-pharmaceuticals-announces-fda-acceptance-companys

3. Ip J, Noseworthy P, Piccini J, et al. Combined Efficacy, Safety, and Test Dose Tolerability of Etripamil for Acute Paroxysmal Supraventricular Tachycardia (PSVT) Across Multiple Clinical Trials. 

. November 3, 2025. Accessed December 9, 2025. 

https://www.ahajournals.org/doi/10.1161/circ.152.suppl_3.4366812

The FDA has approved etripamil (Cardamyst) nasal spray for self-administration in adults with paroxysmal supraventricular tachycardia (PSVT), offering a rapid-acting treatment outside healthcare settings. This novel calcium channel blocker allows patients to manage episodes independently, reducing emergency visits. Clinical trials demonstrated its efficacy, with 59.6% of patients converting to normal sinus rhythm within 30 minutes. The treatment showed mild, transient adverse events and high tolerability. Etripamil's approval marks a significant advancement in PSVT management, providing patients with greater control over their condition.

The FDA has approved etripamil nasal spray for the conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia to sinus rhythm.

Cardiology

Cardiology condition center page is a comprehensive resource for clinical news and insights on cardiovascular and cardiometabolic diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for heart disease and cardiovascular events, as well as associated diabetes, renal failure, and more...

Arrhythmia

FDA News

FDA Approves Etripamil (Cardamyst) Nasal Spray for PSVT

In this survey, investigators evaluated patients’ comprehension of frequently used cutaneous oncology-related words and looked at factors impacting their comprehension.

Key Skin Cancer Terms Not Understood by Almost 1/3 of Patients Surveyed

Alex Hillenbrand is an assistant editor for 

. She earned her BA in English from Lafayette College in 2023 and went on to write for pop culture, entertainment, and sports media outlets before joining MJH Life Sciences in 2025. In her free time, Alex enjoys cooking, going on walks, and meeting new people. Contact her at mjhlifesciences.com.

Cross-sectional analysis from NHANES shows patients with advanced cardiovascular-kidney-metabolic syndrome are at an increased sarcopenia risk. 


Nephrology

The HCPLive nephrology page is a resource for medical news and expert insights on kidney disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments in nephrology, and more.

 Advanced Cardiovascular-Kidney-Metabolic Syndrome Increases Sarcopenia Risk

Chelsie Derman is an associate editor for 

who covers psychiatry, sleep medicine, geriatrics, migraine, and allergy. She joined MJH Life Sciences in September 2023 after graduating from The College of New Jersey with a bachelor’s degree in journalism and professional writing. In her free time, she enjoys creative writing and reading.

Evidence shows fibromyalgia patients face higher overactive bladder rates and symptom severity, highlighting the importance of integrated care.

Rheumatology

Rheumatology condition center page is a comprehensive resource for clinical news and insights on rheumatologic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for arthritis, gout, nr-AxSpA, and more.

Fibromyalgia

On the HCPLive fibromyalgia page, resources on the topics of medical news and expert insight into fibrositis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on fibromyalgia, and more.

High Rates of Overactive Bladder Found in Women With Fibromyalgia

FDA approves zoliflodacin (Nuzolvence) for uncomplicated gonorrhea, offering a new oral treatment amid rising antibiotic resistance.

FDA Approves Zoliflodacin (Nuzolvence) Granules for Uncomplicated Urogenital Gonorrhea

Jain is the medical director of the pediatric and young adult program for hemostasis/thrombosis and sickle cell disease at Loma Linda University Children’s Health.

Jain describes the phase 3 WIL-33 trial, which marked the first test of pdVWF/FVIII in adolescent patients aged <6 years.

Prophylaxis With von Willebrand Factor VIII Reduces Bleeding in Children, With Akshat Jain, MD, MPH

Abigail Brooks is an assistant managing editor for 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

The approval makes inebilizumab-cdon the first and only CD19-targeted B cell therapy indicated for anti-AChR and anti-MuSK Ab+ generalized myasthenia gravis.

Neurology

Neurology condition center page is a comprehensive resource for clinical news and insights on nervous system disorders. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for Alzheimer's disease, brain injury, PTSD, and more.

FDA Approves Inebilizumab-cdon (Uplizna) for Generalized Myasthenia Gravis

Berotralstat (ORLADEYO) oral granules become the first FDA-approved therapy specifically designed to prevent HAE attacks in children aged 2 – 11 years.

FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE  

The approval of a dye- and preservative-free powder formulation of trofinetide offers a new option for adult and pediatric patients with Rett syndrome.

The phase 3 DISCREET study explored the safety and efficacy of apremilast in treating patients with moderate to severe genital psoriasis over 32 weeks.

Plaque Psoriasis

The HCPLive plaque psoriasis page is a resource for medical news and expert insights on psoriasis. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for psoriatic disease, and more.

Apremilast Effective Treatment for Moderate to Severe Genital Psoriasis at 32 Weeks

2025 data show the US is still failing to adequately identify and initiate care for deadly lung disease. Experts discuss what it will take to turn the tide on this public health issue.

This Year In Medicine

COPD

COPD condition center page, resources on the topics of medical news and expert insight into chronic obstructive pulmonary disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on COPD research, treatment, and drug development.

Latest News

Rilzabrutinib Preserves Platelet Response in ITP Through Extension Study, With David Kuter, MD

Dupilumab Shows Greatest Rate of 2-Year Drug Survival Among Atopic Dermatitis Drugs

Rezpegaldesleukin Shows Promise for AD, Comorbid Asthma, With Jonathan Corren, MD

The HCPFive: Top News for Healthcare Providers from the Week of 12/07

FDA Approves Etripamil (Cardamyst) Nasal Spray for PSVT