Chelsie Derman is an associate editor for 

who covers allergy and  psychiatry. 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.

A single dose of lonvoguran ziclumeran (lonvo-z) reduced 

attack rates by 87% compared with placebo in the phase 3 HAELO trial, according to topline 6-month efficacy data released by Intellia Therapeutics.¹

“The results we are seeing from lonvo-z demonstrate its potential to eliminate the need for chronic medication and related challenges,” said HAELO lead investigator Aleena Banerji, MD, professor at Harvard Medical School and director of clinical care at the Center for Drug and Vaccine Allergy at Massachusetts General Hospital, in a statement.1 “If approved as a one-time treatment, I would expect lonvo-z to be an appealing option for many patients.”

HAELO was a randomized, double-blind, placebo-controlled trial evaluating a single 50 mg intravenous dose of lonvo-z, an in vivo CRISPR-based gene editing therapy formerly known as NTLA-2002, in 80 patients aged ≥16 years with type I or II HAE.¹ A total of 52 patients received lonvo-z and 28 received placebo; 71% were receiving long-term prophylaxis (LTP) at baseline and discontinued these therapies before dosing.¹ The primary endpoint assessed the mean monthly attack rate during weeks 5 through 28.

During the 6-month efficacy period, the mean monthly attack rate was 0.26 in the lonvo-z arm compared with 2.10 in the placebo arm (

 <.0001), meeting the primary endpoint.¹ All key secondary endpoints were also met, including a 62% rate of patients who were both attack-free and LTP-free, compared with 11% in the placebo group (

 <.0001).¹ At data cutoff, all patients treated with lonvo-z, including those who crossed over after week 28, remained off prophylactic therapy.¹

Safety findings were consistent with earlier-stage data. The most common treatment-emergent adverse events, all reported as mild or moderate, included infusion-related reactions, headache, and fatigue. No serious adverse events were observed in the treatment arm during the primary observation period.¹

HAE, a condition characterized by recurrent, potentially life-threatening angioedema attacks driven by dysregulated bradykinin production, remains a rare genetic disorder affecting approximately 1 in 50,000 individuals.² Despite the availability of targeted therapies, including plasma kallikrein inhibitors such as lanadelumab and berotralstat, management often requires chronic administration and does not fully eliminate breakthrough attacks.³

⁴ The need for frequent injections or daily oral therapy contributes to treatment burden and adherence challenges.

Lonvo-z is designed to address this unmet need through in vivo gene editing. Using CRISPR/Cas9 technology, the therapy targets and inactivates the KLKB1 gene in hepatocytes, reducing kallikrein production and downstream bradykinin generation.¹ This mechanism builds on established clinical validation of kallikrein inhibition in HAE but introduces the possibility of sustained effect after a single administration.

The program has received multiple regulatory designations, including Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the US Food and Drug Administration, as well as PRIME designation in the European Union.¹ Intellia has initiated a rolling biologics license application (BLA) submission, with completion anticipated in the second half of 2026 and potential US launch in 2027 if approved.

Although the magnitude of effect observed in HAELO is notable, interpretation remains constrained by the limited follow-up duration of 6 months for the primary endpoint. Longer-term durability, potential delayed adverse effects, and real-world generalizability remain key unanswered questions, particularly given the permanent genomic modification underlying the therapy. In addition, the trial required discontinuation of baseline prophylaxis before dosing, which may not fully reflect clinical practice patterns.

If confirmed and approved, lonvo-z would represent a new therapeutic paradigm in HAE, shifting from chronic suppression of disease pathways to potentially durable modification of the underlying biology.1 However, clinicians will likely weigh efficacy against uncertainties related to long-term safety, reversibility, and patient selection in considering integration into practice.

“As the first phase 3 data reported for an in vivo gene editing therapy, today’s HAELO results represent a profound milestone for Intellia, the broader CRISPR and precision medicine fields, and most importantly, the HAE community,” said Intellia President and Chief Executive Officer John Leonard, MD, in the press release.

“These data affirm lonvo-z’s potential, with one dose, to offer prolonged freedom from both attacks and the need for ongoing therapy.”

Intellia Therapeutics. Intellia Therapeutics reports positive phase 3 results in hereditary angioedema, marking a global first for in vivo gene editing. April 27, 2026. 

https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-reports-positive-phase-3-results

Learn About Hereditary Angioedema. DiscoverHAE. 

https://www.discoverhae.com/what-is-hereditary-angioedema

Lanadelumab Cuts Monthly HAE Attack Rates in Real-World 3-Year Study. HCPLive. Published on July 22, 2025. Accessed April 27, 2026. 

https://www.hcplive.com/view/lanadelumab-cuts-monthly-hae-attack-rates-in-real-world-3-year-study

Derman C. FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE. HCPLive. Published on December 12, 2026. Accessed April 27, 2026. 

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

Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for Lonvoguran Ziclumeran (lonvo-z) as a One-Time Treatment for Hereditary Angioedema - Intellia Therapeutics. Intellia Therapeutics. Published on April 27, 2026. Accessed April 27, 2026. 

https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-initiates-rolling-submission-biologics

Articles

Phase 3 HAELO topline data show a single 50 mg IV dose of lonvoguran ziclumeran achieved an 87% reduction in mean monthly hereditary angioedema attack rates versus placebo through weeks 5–28 (0.26 vs 2.10; P<.0001). Most participants discontinued baseline long-term prophylaxis pre-dose, and 62% became both attack-free and prophylaxis-free versus 11% with placebo. Safety was consistent with prior studies, with mainly mild-to-moderate infusion reactions, headache, and fatigue, and no treatment-arm serious adverse events in the primary period.

Phase 3 HAELO data show significant reductions in attack rates and treatment burden with a single-dose in vivo CRISPR therapy; BLA submission underway with FDA.

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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.

Single-Dose Lonvoguran Ziclumeran Cuts HAE Attacks by 87% in Phase 3 Trial

Michelle Bichchau Nguyen, MD, MBA, MPH, is an associate professor of dermatology at Tufts University School of Medicine and the director of Mohs Micrographic Surgery.


Dermatologic surgeries require clear communication and guidance by those performing the procedure, with patient satisfaction being an invaluable consideration in such circumstances.

A positive experience among patients treated in outpatient dermatology clinics is not only based on rapport with clinicians but also on such elements as optimal utilization of space, the support of staff members, and optimal wait times. In a review of best practices related to patient experience, Michelle Nguyen, MD, MBA, MPH, associate professor of dermatology at Tufts University School of Medicine and director of Mohs Micrographic Surgery, touched on this topic in an interview following a session presented at the 

2026 American Academy of Dermatology (AAD) Annual Meeting

The following Q&A interview transcript summarizes her responses to inquiries by 

 In your talk, you gave several pearls to improve patient satisfaction and enhance patient experience in dermatology. What were the notable takeaways from your portion of this session regarding surgery?

Correct, and I'm currently the director of dermatology surgery at Tufts Medical Center, which is part of Tufts University School of Medicine. So my portion covered some of the top tips for providing the optimal patient experience for people undergoing surgical procedures. And you know, we always think that it's the surgical outcomes that matter the most for patients, like how things look, making sure there are no complications, and, obviously, that's very important.

But what patient care even more than that is how they are treated before, during, and after the visit. Clear explanation of what the procedure is, a detailed, step-by-step, explanation, giving them the opportunity to ask questions, raise their concerns, and set realistic expectations [is key]. There are no scarless surgeries. We always tell patients that we use the best techniques. But [then we say] here are the things that you can do at home to help your scars look better and in the in the future, if you have any concerns at all about how your scar looks or feel, we're happy to see you back anytime, and patients are really comforted by knowing that you're there for them throughout the surgical procedure, but also the post op care and beyond.

So having that relationship that extends beyond the actual procedure is really important, and having well-trained staff that can comfort and care for the patient, as you're doing the procedures, answering common patient questions, and doing patient education when you're not there are also a very key part of that optimal patient experience for surgery.

These do sound like important strategies for improving patient experiences, especially with regard to your comments about staff guidance.

Yes, and you know, people underestimate the role of staff in crafting that optimal surgical experience, because our staff actually spends way more time with the patients than we do. Oftentimes, our surgical procedures take is actually a very small part of that care experience. So you should treat your staff well, train them well. Empower them to help patients whenever they can, without your input.

If you were looking at only one of these pearls you provided, what would you say you've seen in your practice that has really worked for patients?

We like to give information. We like to educate, and we think that the more we talk, the more information we're providing, but that might not be the information that the patient is looking for. I think a key part of crafting the optimal patient experience is actually asking the patient what their concerns are, what they worry about in the aftercare, and then actually hearing about their concerns and finding ways to address them. So, my number one tip is to ask and listen.

How much does clear communication about treatment expectations influence patient satisfaction, especially for chronic skin conditions?

It’s hugely important because when there's a mismatch between what the patient expects and reality, that is when the patient gets unhappy. Part of our job as clinicians, as with educators, is to set realistic expectations, especially in this age of social media and ChatGPT, where people get information from a very diverse range of sources of information. Some are correct, and some are not.

You just want to be clear that this is a chronic condition, we will manage it for you. These are the options. These are the escalating treatments that we will try. And it takes time. Oftentimes, these are things that you struggle with for years. It's going to take us a bit of time to put you on the right regimen, for your skin condition, for your quality of life, for your goals of care, and so establishing that treatment plan and milestones ahead really helps set expectations so that the patients feel like you understand and that you're there for them for the long term.

What would you say is the biggest driver of patient dissatisfaction in dermatology practices?

 We are a high-volume practice. We see many patients in a relatively short period of time. Therefore, it is very crucial for us to take the time to make patients feel like they are listened to, and that we are spending adequate time to provide the care and listen to them. I always tell my colleagues to sit down. You know, just the fact that you're sitting down and having a conversation instantly makes patients feel like you are spending the time, and it's worth it to wait three months to see you right, and paying the co-pays and all of that. Establishing that rapport and sitting down and giving the patient the time and space to talk and express that concern is very important.

What would you what advice would you give to an early-career dermatologist who wants to build a more patient-centered practice?

To build on what I said about listening to the patient, don't take it personally. You know, when we were first starting out, whenever we have a suboptimal patient review, it really hurt. You take it as a bad note on your performance and who you are as a clinician, and your worth as a doctor. I would say that's not true, right?

Patients respond because they're hurt before they felt let down, and some of those words can hurt because we're people too, but don't let don't take that so personally. It's a learning opportunity, and you try to take every negative comment as a small grain of truth. You should focus on that grain of truth; you get better. And eventually, you find that your interactions with patients just automatically get better over time.

The quotes contained in this summary were edited for the purposes of clarity.

Nguyen had no relevant financial disclosures of note.

Nguyen M, et al. U005 Top Tips from Top Docs: Pearls to Improve Patient Satisfaction and Enhance Patient Experience. Session presented at: 2026 American Academy of Dermatology Annual Meeting; March 27–31, 2026; Denver, CO.

Queen D, Trager MH, Samie FH, et al. Patient Satisfaction of General Dermatology Providers: A Quantitative and Qualitative Analysis of 38,008 Online Reviews. JID Innov. 2021 Aug 19;1(4):100049. doi: 10.1016/j.xjidi.2021.100049. PMID: 34909746; PMCID: PMC8659734.

Optimizing dermatologic surgery experience depends as much on process and communication as on clinical outcomes. Pre-, intra-, and post-procedure counseling should include stepwise explanations, opportunities for questions, and realistic expectations, including unavoidable scarring and practical scar-optimization strategies. Longitudinal availability for postoperative concerns reassures patients and reinforces trust. Staff training and empowerment are central, given their disproportionate patient-facing time. In high-volume settings, perceived attentiveness—asking about patient concerns, actively listening, and even sitting during conversations—mitigates dissatisfaction and strengthens rapport.

In this interview, Nguyen discusses improving patient experiences during surgical procedures in dermatology practices, such as Mohs surgery.

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.

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Q&A: Tips to Improve Dermatologic Surgery Experience, With Michelle Bichchau Nguyen, MD, MBA, MPH

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.

A supplemental New Drug Application (sNDA) has been submitted to the US Food and Drug Administration (FDA) for the broadening of roflumilast cream 0.05% (Zoryve) to treat mild to moderate atopic dermatitis in infants down to the age of 3 months.

The sNDA submission, announced on April 27 by Arcutis Biotherapeutics, was backed by data from a pair of open-label studies assessing once-daily roflumilast cream 0.05% in infants aged 3 to less than 24 months with mild to moderate atopic dermatitis. The disease itself is the most prevalent form of eczema in the US, impacting an estimated 9.6 million children and 16.5 million adults.

“Atopic dermatitis often begins within the first year of life, yet treatment options for infants are extremely limited,” Mercedes E. Gonzalez, MD, medical director of Pediatric Skin Research, LLC and INTEGUMENT-INFANT clinical trial investigator, said in a statement.

Parents and caregivers are often faced with difficult decisions around managing these symptoms, including concerns about the long-term use of topical steroids. Because their immune system and skin barrier are still developing, infants are particularly vulnerable to the burdensome symptoms of atopic dermatitis, including visible disease and intense itch that can disrupt sleep and significantly impact both infants and their caregivers.”

The disease often manifests during infancy, imposing considerable burden on both patients and their caregivers. The disease presents unique treatment considerations among infants, including a higher body surface area-to-body mass ratio heightening systemic exposure risk from topical medications.

The data leading to this sNDA first included a phase 1 pharmacokinetic study in 19 infants demonstrating a safety, efficacy, and pharmacokinetic profile over 4 weeks. These findings aligned with data from prior research in older pediatric populations. In the larger phase 2 INTEGUMENT-INFANT trial, 101 infants were recruited and roflumilast cream’s safety, tolerability, and efficacy were evaluated across 4 weeks of treatment.

The medication was shown by Gonzalez and colleagues to be well tolerated, with patients’ most commonly reported adverse events (AEs) being nasopharyngitis, diarrhea, upper respiratory tract infection, and vomiting.

 These AEs were described as consistent with the broader INTEGUMENT program experience.

In the infants completing the full 4-week treatment course (n=96), investigators found 34.4% attained success in their Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD) score, defined as a ‘clear’ or ‘almost clear’ score with at least a 2-grade improvement from baseline.

 The team further noted 49% succeeded in achieving a vIGA-AD score at the 4-week mark.

Improvements were observed by Gonzalez et al as early as Week 1. Among infants showing scalp involvement at baseline, 67.5% saw vIGA-scalp success at the 4-week mark, and 58.3% attained an EASI-75 score at Week 4.

 The team further noted notable caregiver-reported itch outcomes, including that 72.7% of infants met the threshold for Worst Scratch Itch Numeric Rating Scale success at Week 4. Rapid onset was observed in these infants, as suggested by a 46.6% response rate on the Dynamic Pruritus Scale by 10 minutes post-application.

Roflumilast cream 0.05% is already FDA-approved for mild to moderate AD in patients aged 2 to 5 years.

 This new move could expand its indication if it receives approval by FDA officials for this newer age range.

“Safe and effective long-term therapies suitable for use anywhere on the body, including sensitive regions such as the face and skin folds, remain an important unmet need for infants with atopic dermatitis,” Gonzalez said in her statement.

 “If approved, [roflumilast] cream 0.05% could offer a much-needed, non-steroidal treatment option developed specifically for very young children, including infants.”

Arcutis Submits Supplemental New Drug Application to the FDA for ZORYVE® (roflumilast) Cream 0.05% to Expand Indication for Treatment of Atopic Dermatitis to Infants Down to 3 Months. Arcutis Biotherapeutics, Inc. April 27, 2026. 

https://investors.arcutis.com/news-releases/news-release-details/arcutis-submits-supplemental-new-drug-application-fda-zoryver

Smith T. FDA Approves Roflumilast (Zoryve) Cream 0.05% for Atopic Dermatitis in Children Aged 2-5 Years. HCPLive. October 6, 2025. Accessed April 27, 2026. 

https://www.hcplive.com/view/fda-approves-roflumilast-zoryve-cream-0-05-for-atopic-dermatitis-in-children-aged-2-5-years

Arcutis submitted an sNDA to expand roflumilast cream 0.05% for mild-to-moderate atopic dermatitis to infants aged ≥3 months. The filing is supported by open-label phase 1 PK (n=19) and phase 2 INTEGUMENT-INFANT (n=101) data over 4 weeks, addressing infant-specific exposure concerns. Treatment was well tolerated, with AEs including nasopharyngitis, diarrhea, URTI, and vomiting. Efficacy signals included vIGA-AD success in 34.4% (n=96), early improvement by Week 1, EASI-75 in 58.3%, and rapid caregiver-reported itch relief.

This supplemental New Drug Application for roflumilast cream (Zoryve) to the FDA was supported by the INTEGUMENT-INFANT open-label study.

FDA News

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.

Roflumilast Cream 0.05% sNDA Submitted to FDA for Infants Down to 3 Months with Atopic Dermatitis

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 US Food and Drug Administration (FDA) has approved anifrolumab-fnia (Saphnelo) for subcutaneous self-administration via a once-weekly 120 mg autoinjector, the Saphnelo Pen, for adult patients with moderate to severe systemic lupus erythematosus (SLE) receiving standard therapy.¹ 

According to AstraZeneca, the approval was based on results from the phase III TULIP-SC trial, published in 

 in January 2026.² Anifrolumab had previously been available only as an intravenous infusion administered in clinical settings since its initial US approval in 2021.

What is anifrolumab (Saphnelo) approved for? 

Anifrolumab is approved for adult patients with moderate to severe SLE receiving standard therapy, now available as both an IV infusion and a once-weekly 120 mg subcutaneous autoinjector (Saphnelo Pen).

Anifrolumab is a fully human monoclonal antibody binding subunit 1 of the type I interferon receptor, blocking the activity of type I interferons such as IFN-alpha, IFN-beta, and IFN-kappa implicated in SLE pathogenesis.

The phase 3 TULIP-SC trial demonstrated a statistically significant and clinically meaningful reduction in SLE disease activity at week 52 on the BICLA response endpoint with subcutaneous anifrolumab versus placebo, with 29.0% of anifrolumab-treated patients achieving DORIS remission.

"The approval of anifrolumab as a self-administered autoinjector is exciting news as it makes this important medicine more convenient and accessible for many more patients,” Susan Manzi, MD, MPH, chair of the Allegheny Health Network Medicine Institute, director of the Lupus Center of Excellence at the AHN Autoimmunity Institute, and principal investigator of TULIP-SC, said in a statement. “With its proven ability to significantly reduce disease activity and the risk of organ damage, anifrolumab has been a much-needed innovation in lupus."

The subcutaneous formulation addresses a practical barrier for patients requiring every-four-week IV infusions in clinic and aligns with recent ACR and EULAR guideline updates emphasizing treat-to-target strategies and corticosteroid minimization in SLE.¹ 

Anifrolumab remains the only approved type I interferon receptor antagonist in SLE, and AstraZeneca reports > 40,000 patients globally have received the drug to date.¹ Subcutaneous administration of anifrolumab has already been approved in the EU and Japan.¹

TULIP-SC Trial Design and Primary Efficacy Results

The TULIP-SC trial was a phase 3, multicenter, randomized, double-blind, placebo-controlled study evaluating subcutaneous anifrolumab versus placebo in adults aged 18 to 70 years with moderate to severe SLE on standard therapy, including oral corticosteroids, antimalarials, and/or immunosuppressants.¹ A total of 367 participants were randomly assigned in a 1:1 to receive anifrolumab 120 mg or placebo weekly via a pre-filled, single-use syringe.¹

The primary endpoint was British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) response at week 52, which requires improvement in all organ domains with active disease at baseline and no new flares.¹ According to AstraZeneca, subcutaneous anifrolumab achieved a statistically significant and clinically meaningful reduction in disease activity compared with placebo on this endpoint.¹ A planned interim analysis was conducted when the first 220 participants reached week 52 or withdrew from the study.¹

The press release did not disclose exact BICLA response rates, effect sizes, or p-values for the primary endpoint. Full trial data were published by Manzi et al in Arthritis & Rheumatology.²

Anifrolumab Subcutaneous Safety Profile and Secondary Endpoints in TULIP-SC

Across pre-specified secondary and exploratory endpoints, anifrolumab demonstrated clinically meaningful effects on multiple outcome measures, according to AstraZeneca.¹ The drug reduced SLE disease activity while enabling corticosteroid tapering to low doses (≤7.5 mg/day prednisone equivalent), and more patients receiving anifrolumab achieved BICLA response sooner compared with placebo.¹ Time to first flare was numerically delayed in the anifrolumab arm.¹

Among patients receiving anifrolumab, 29.0% achieved DORIS remission and 40.1% attained low-level disease activity as measured by the Lupus Low Disease Activity State (LLDAS) score on pre-specified secondary and exploratory endpoints.¹ The DORIS remission criteria include a clinical SLEDAI-2K score of 0, physician global assessment below 0.5, and prednisone-equivalent dose of 5 mg/day or less on stable immunosuppressant doses.¹

The safety profile observed with subcutaneous anifrolumab was consistent with the known clinical profile of the IV formulation, per the company.¹ The most common adverse reactions (≥5% incidence) with anifrolumab include nasopharyngitis, upper respiratory tract infections, bronchitis, infusion-related reactions, herpes zoster, and cough.¹ Serious infections, hypersensitivity reactions including anaphylaxis, and herpes zoster remain adverse events of special interest; the label notes increased malignancy risk with immunosuppressants and recommends avoiding live vaccines.¹

Anifrolumab continues to be evaluated in phase 3 trials across 4 additional type I interferon-driven indications, including cutaneous lupus erythematosus (LAVENDER), idiopathic inflammatory myopathies (JASMINE), systemic sclerosis (DAISY), and lupus nephritis (IRIS).¹ The IV formulation remains the first biologic with 4-year placebo-controlled remission data in SLE from the TULIP-LTE trial.¹

AstraZeneca. SAPHNELO approved in the US for subcutaneous self-administration as a new autoinjector for the treatment of systemic lupus erythematosus. Published April 27, 2026. Accessed April 27, 2026. 

https://www.businesswire.com/news/home/20260427513110/en/SAPHNELO-approved-in-the-US-for-subcutaneous-self-administration-as-a-new-autoinjector-for-the-treatment-of-systemic-lupus-erythematosus

Manzi S, Furie R, et al. Efficacy and safety of subcutaneous anifrolumab in systemic lupus erythematosus: the randomized, phase 3, TULIP-SC study. Arthritis Rheumatol. 2025. doi:10.1002/art.70041

Morand E, Furie R, Tanaka Y, et al. Trial of anifrolumab in active systemic lupus erythematosus. N Engl J Med. 2020;382(3):211-221.

FDA authorized once-weekly 120 mg subcutaneous anifrolumab autoinjector self-administration for adults with moderate-to-severe SLE on standard therapy, expanding access beyond q4-week IV infusions. Approval was supported by phase III TULIP-SC (n=367), a randomized, double-blind, placebo-controlled study meeting its week-52 BICLA primary endpoint. Secondary analyses showed corticosteroid tapering to ≤7.5 mg/day, earlier responses, numerically delayed flares, 29% DORIS remission, and 40.1% LLDAS. Safety mirrored IV labeling, with infection, hypersensitivity, and herpes zoster risks. It remains the only approved type I interferon receptor antagonist, with phase III studies in other interferon-driven diseases.

The FDA approved anifrolumab via a once-weekly subcutaneous autoinjector for moderate to severe SLE based on phase 3 TULIP-SC trial results.

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.

Anifrolumab (Saphnelo) Wins FDA Approval for Subcutaneous Self-Administration in SLE

Patrick Campbell is the editorial director of HCPLive. Patrick has spent years spearheading coverage surrounding cardiometabolic health and rheumatic disease for MJH Life Sciences. Before joining MJH Life Sciences in 2019, he spent time as a beat reporter and/or multimedia specialist with the Pocono Record, Star News Group, and NJ Advance Media. He is the executive producer for multiple HCPLive podcasts, including Diabetes Dialogue, Don't Miss a Beat, Kidney Compass, Medical Ethics Unpacked, The Medical Sisterhood, and Skin of Color Savvy. 


Follow him on Twitter @RealPatCampbell or reach him via email at PCampbell@mjhlifesciences.com.

 has approved a supplemental New Drug Application (sNDA) for lumateperone (Caplyta) based on long-term data supporting the prevention of relapse in adults with 

Johnson & Johnson announced the lumateperone FDA approval on April 26, 2026. The approval stems from a phase 3 randomized withdrawal trial demonstrating a 63% reduction in relapse risk versus placebo over a 26-week double-blind treatment period.¹

"This label update, backed by long-term Phase 3 data demonstrating a significant delay in time to relapse, reinforces our commitment to advancing evidence-based therapies to support each patient's individual needs including a proven therapy supporting stability over time," said Celine Goldberger, MD, PhD, Vice President of Global Medical Affairs, Neuroscience, Innovative Medicine at Johnson & Johnson.

Lumateperone is already approved in adults for schizophrenia, as adjunctive therapy with antidepressants for major depressive disorder, and for depressive episodes associated with bipolar I or II disorder. The sNDA expands the label by incorporating long-term efficacy and tolerability data reinforcing its role in sustained disease management, an area of particular clinical interest given the frequency of relapse events in this population.

Lumateperone (Caplyta) is FDA-approved in adults for schizophrenia, as adjunctive therapy with antidepressants for major depressive disorder, and for depressive episodes associated with bipolar I or II disorder as monotherapy or adjunctive therapy with lithium or valproate. The updated label now includes long-term data supporting relapse prevention in schizophrenia.

Lumateperone is characterized by antagonist activity at central serotonin 5-HT2A receptors and partial agonist activity at central dopamine D2 receptors, with high 5-HT2A occupancy and moderate D2 occupancy at therapeutic doses.

In a phase 3 randomized withdrawal trial, lumateperone 42 mg significantly extended time to relapse versus placebo over 26 weeks (

=.0002), reducing relapse risk by 63% (HR, 0.37), with 84% of patients remaining relapse-free at six months.

Lumateperone efficacy in the Study 304 randomized withdrawal trial

Study 304 was a multicenter, multinational, double-blind, placebo-controlled, randomized withdrawal study enrolling adults with schizophrenia.¹ The trial included an 18-week open-label phase during which patients received lumateperone 42 mg once daily. Patients meeting predefined stabilization criteria were randomized to continue lumateperone 42 mg (N=110) or switch to placebo (N=114) for up to 26 weeks.¹

On the primary endpoint, lumateperone significantly extended time to first symptom relapse compared with placebo during the double-blind phase (P = .0002).¹ Patients receiving lumateperone had a 63% lower risk of relapse versus placebo (hazard ratio, 0.37), and 84% of lumateperone-treated patients remained relapse-free over 6 months.¹ The key secondary endpoint, time to all-cause treatment discontinuation including relapse, also favored lumateperone, with a significant delay compared with placebo.¹

"These Phase 3 results, showing significantly longer time to relapse with 84% remaining relapse free over 6 months, provide clinicians with another tool offering long-term stability for people living with schizophrenia," said Christoph U. Correll, MD, clinical professor of psychiatry at the Zucker School of Medicine at Hofstra/Northwell, New York.

Lumateperone safety profile and metabolic outcomes in long-term use

The safety profile of lumateperone in Study 304 remained consistent with the existing body of clinical data, and no new safety concerns were identified, according to Johnson & Johnson.¹ The most common treatment-related adverse event was headache, occurring in at least 5% of patients and at least twice the rate observed with placebo.¹ No clinically relevant increases in prolactin or cardiometabolic parameters emerged at the end of the double-blind treatment period.¹

Data from a separate 12-month open-label extension study in schizophrenia further supported long-term tolerability. Patients treated with lumateperone experienced a mean weight change of −2.05 kg (−4.52 lbs) over one year, with sustained improvements or stability in metabolic parameters.¹ In prior short-term clinical studies, lumateperone was similar to placebo in weight change, metabolic effects, and extrapyramidal symptoms.¹

Lumateperone is also being evaluated in clinical studies for other neuropsychiatric and neurological conditions beyond its current FDA-approved indications, according to the company.¹

Johnson & Johnson. FDA approves CAPLYTA (lumateperone) sNDA with robust new data supporting reduced risk of relapse in schizophrenia. Published April 26, 2026. Accessed April 26, 2026. 

https://www.prnewswire.com/news-releases/fda-approves-caplyta-lumateperone-snda-with-robust-new-data-supporting-reduced-risk-of-relapse-in-schizophrenia-302753851.html

Intra-Cellular Therapies. Announces positive topline results in phase 3 trial evaluating CAPLYTA for the prevention of relapse in patients with schizophrenia. Published November 5, 2024. Accessed April 26, 2026. 

https://www.globenewswire.com/news-release/2024/11/05/2974784/30597/en/Intra-Cellular-Therapies-Announces-Positive-Topline-Results-in-Phase-3-Trial-Evaluating-CAPLYTA-for-the-Prevention-of-Relapse-in-Patients-with-Schizophrenia.html

FDA clearance of a lumateperone sNDA adds phase 3 long-term evidence for relapse prevention in adult schizophrenia. In the multinational Study 304 randomized-withdrawal design, stabilized patients continuing lumateperone 42 mg daily showed significantly prolonged time to symptom relapse versus placebo (HR 0.37; P=.0002), with 84% remaining relapse-free through 26 weeks. Treatment also delayed all-cause discontinuation. Safety remained consistent with prior experience, without clinically meaningful prolactin or cardiometabolic changes, and 12-month open-label data showed modest mean weight reduction with metabolic stability.

The FDA approved a lumateperone sNDA adding relapse prevention data in schizophrenia based on a 63% reduction in relapse risk versus placebo.

Schizophrenia

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

Major Depressive Disorder

Major Depressive Disorder condition center page, resources on the topics of medical news and expert insight into depression can be found. Content includes articles, interviews, videos, podcasts, and breaking news on MDD research, treatment, and drug development.

Psychiatry

Psychiatry condition center page is a comprehensive resource for clinical news and insights on mental illnesses. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for depression, schizophrenia, bipolar disorder, anxiety, and more.

Lumateperone sNDA Approved with Relapse Prevention Data in Schizophrenia

AbbVie submitted an FDA application for risankizumab subcutaneous induction in Crohn's disease based on phase 3 AFFIRM data.

Gastroenterology

Gastroenterology condition center page is a comprehensive resource for clinical news and insights on digestive and GI conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for 

, IBS and IBD, Crohn's disease, and more.

Inflammatory Bowel Disease

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

Risankizumab (Skyrizi) SC Induction Application Submitted to FDA for Crohn's disease

Andrea Cipriani, MD, PhD, from the University of Oxford.

Videos

In a randomized trial, PETRUSHKA improved antidepressant continuation and 24-week symptom outcomes by integrating clinical data with patient preferences.

PETRUSHKA Tool Improves Antidepressant Continuation, With Andrea Cipriani, MD, PhD

Armstrong spoke in an interview regarding several different sessions presented at the 2026 AAD meeting.

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.

Q&A: IL-23, Oral Therapies, and Inflammatory Disease Management, With April W. Armstrong, MD, MPH

Eingun James Song, MD, is the director of clinical research for Frontier Dermatology. 

OX40 for Atopic Dermatitis: A Target at a Crossroads

David Kass, MD, is the Abraham and Virginia Weiss Professor of Cardiology at the Johns Hopkins University School of Medicine.

Kass explains 2 distinct biological subgroups discovered in HFpEF and how they relate to a paradigm shift. 

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...

Heart Failure

The HCPLive heart failure page is a resource for medical news and expert insights on HF. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for heart disease, reduced and preserved ejection fraction, and more.

Obesity Management

Obesity Management condition center page, resources on the topics of medical news and expert insight into clinical weight management can be found. Content includes articles, interviews, videos, podcasts, and breaking news on obesity research, treatment, and drug development.

Severe Obesity Linked to Distinct Myocardial Dysfunction in HFpEF

Diana Isaacs, PharmD, is an endocrine clinical pharmacist, director of Education and Training in Diabetes Technology, and codirector of Endocrine Disorders in Pregnancy at the Cleveland Clinic.

Podcasts

Elizabeth Bauer, MD, joins the show to discuss her AACE 2026 presentation on key updates in obesity management and nutrition.

Endocrinology

The HCPLive endocrinology page is a comprehensive resource for clinical news and insights on endocrine system conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for diabetes, hormonal disease, and more.

Type 1 Diabetes

Type 2 Diabetes

Strategic Alliance Partnership

Diabetes Dialogue: Nutrition, Medication, and Treatment in Obesity With Elizabeth Bauer, MD

Auchus discusses the 2-year results from CAHtalyst Adult, highlighting the substantial retention rates over the open-label extension period.

Crinecerfont Reduces Glucocorticoid Doses in CAH, With Richard Auchus, MD, PhD

Jagbir Gill, MD, MPH, is an Associate Professor of Medicine in the Division of Nephrology at the University of British Columbia and Medical Director of the Kidney Transplant Program at St. Paul’s Hospital in Vancouver. He also serves as Medical Director of Data and Quality for BC Transplant, President of the Canadian Organ Replacement Register Board of Directors, and as Treasurer for the Canadian Society of Transplantation.

Gill describes how storytelling and patient representation can build trust and education in Indigenous populations regarding transplantation. 


Chronic Kidney Disease

Transplant Care Strategies to Target Mistrust, Close Gaps in Indigenous Communities

Scott Isaacs, MD, is an Adjunct Assistant Professor of Medicine at Emory University. He currently serves as Immediate Past President of the American Association of Clinical Endocrinology (AACE).

Isaacs explains how prediabetes care is shifting toward early intervention, risk stratification, and prevention of cardiometabolic disease, not just progression to diabetes.

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