Efzofitimod did not significantly reduce the mean daily oral corticosteroid (OCS) dose after 48 weeks in people with 

 sarcoidosis although clinical benefits were observed. Accordingly, aTyr Pharma’s Phase 3 EFZO-FIT evaluating the therapy did not meet its primary endpoint.

“The results of EFZO-FIT™ demonstrate not only strong evidence of clinical efficacy for efzofitimod’s effect on quality of life and its capacity to facilitate steroid withdrawal, but also increased confidence that steroids overall can be reduced to manage symptoms and lung function without the fear of worsening disease,” principal investigator Daniel Culver, DO, Chair of the Department of Pulmonary Medicine at the Cleveland Clinic, said in a statement.

 “This trial represents an important step forward for the field, as it is by far the largest interventional study completed in sarcoidosis to date, demonstrating the feasibility of conducting a global multi-center randomized controlled trial. The findings provide valuable insights about the treatment of pulmonary sarcoidosis, which are likely to inform treatment practices in the future.”

EFZO-FIT was a global interventional study sarcoidosis that compared the efficacy and safety of efzofitimod at 3.0 mg/kg and 5.0 mg/kg doses versus placebo after 48 weeks of treatment in 268 patients with pulmonary sarcoidosis. The trial protocol included a guided steroid taper in the first 12 weeks of the study, followed by continued taper or rescue until week 48.

Study investigators found that the 5.0 mg/kg efzofitimod group had a mean reduction to 2.79 mg in mean daily OCS dose compared to 3.52 in the placebo group (

= .3313). All further statistical testing is reported as nominal findings as the study’s statistical analysis plan was designed on a hierarchical assessment basis.

Investigators did find a clinical improvement in the change from baseline on King’s Sarcoidosis Questionnaire (KSQ)-Lung score at week 48 for 5.0 mg/kg efzofitimod (10.36) compared to placebo (6.19; 

= .0479). Furthermore, 46.9% of patients on 5.0 mg/kg of efzofitimod achieved complete steroid withdrawal with stable KSQ-Lung score compared to 35.7% on placebo (

Furthermore, patients who achieved complete steroid withdrawal at week 48 with 5.0 mg/kg efzofitimod had greater improvements in KSQ-Lung compared to placebo (

= .0199). Overall, 52.6% of patients treated with 5.0 mg/kg efzofitimod achieved complete steroid withdrawal compared with 40.2% on placebo (

Forced vital capacity (FVC) values at week 48 were maintained.The change from baseline in absolute percent predicted FVC was -1.81 for patients in the 5.0 mg/kg efzofitimod group compared to 2.11 in the placebo group (

“This study demonstrates that patients with chronic, symptomatic sarcoidosis can be managed with substantially lower steroid doses than previously thought. In spite of a higher than anticipated placebo response, we found that treatment with efzofitimod was associated with a greater amount of steroid reduction and an improvement in the KSQ-Lung score,” Sanjay S. Shukla, MD, MS, President and Chief Executive Officer of aTyr Pharma, added.

 “These consistent findings suggest that treatment with efzofitimod may positively impact quality of life and preserve lung function while reducing steroid burden, providing a meaningful benefit to patients. We look forward to discussing the totality of the data with the FDA in order to determine the path forward for efzofitimod in pulmonary sarcoidosis, as there remains an urgent need for a safe and effective treatment option to address the unmet needs of this underserved population.”

Efzofitimod was generally well-tolerated at both the 3.0 mg/kg and 5.0 mg/kg doses, with a safety profile consistent with that seen in other trials of the therapy. Further data on efzofitimod will be presented at the upcoming European Respiratory Society Congress on September 30, in Amsterdam, Netherlands. aTyr Pharma plans to engage with the United States Food and Drug Administration (FDA) to review the data and determine the path forward for efzofitimod in pulmonary sarcoidosis.

In June 2025, aTyr Pharma announced positive early data in people with diffuse and limited 

systemic sclerosis-related interstitial lung disease

 (SSc-ILD) from the ongoing Phase 2 EFZO-CONNECT study. Investigators found that all patients had stable or improved modified Rodnan Skin Score (mRSS) and 3 of 4 evaluable, efzofitimod-treated patients with diffuse SSc-ILD had at least a 4-point improvement.

aTyr Pharma Announces Topline Results from Phase 3 EFZO-FIT™ Study of Efzofitimod in Pulmonary Sarcoidosis. News release. aTyr Pharma. September 15, 2025. 

https://finance.yahoo.com/news/atyr-pharma-announces-topline-results-113000869.html

Johnson V. Efzofitimod Shows Promising Early Efficacy in Systemic Sclerosis-Related Interstitial Lung Disease. Article. HCPLive. June 5, 2025. 

https://www.hcplive.com/view/efzofitimod-shows-promising-early-efficacy-in-systemic-sclerosis-related-interstitial-lung-disease

Articles

Efzofitimod did not significantly reduce the mean daily oral corticosteroid dose in pulmonary sarcoidosis patients after 48 weeks, failing to meet the primary endpoint of the Phase 3 EFZO-FIT study. However, clinical benefits were observed, including improved quality of life and steroid withdrawal facilitation. The study demonstrated the feasibility of conducting large-scale trials in sarcoidosis and provided insights into treatment practices. Efzofitimod was well-tolerated, and further data will be presented at the European Respiratory Society Congress. aTyr Pharma plans to discuss the findings with the FDA.

Efzofitimod Fails to Meet Primary End Point in Phase 3 Pulmonary Sarcoidosis Trial

COLLIGO-HCM: Mavacamten (CAMZYOS) Effective in Obstructive HCM With Pankaj Arora, MD

Results from the COLLIGO-HCM observational study have indicated the safety and efficacy of mavacamten (CAMZYOS) in patients with symptomatic 

obstructive hypertrophic cardiomyopathy (HCM)

, which is consistent with prior studies.

Mavacamten is the first and only cardiac myosin inhibitor approved by the US Food and Drug Administration for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive HCM.

COLLIGO-HCM is part of a larger program by parent company Bristol Myers Squibb, titled Worldwide AnalYsis on eFfectiveness AcRoss divErse populations for Real-world mavacamten use in patients with Hypertrophic CardioMyopathy (WAYFARER-HCM), which currently spans 7 countries and includes >3000 patients. It aims to investigate the effectiveness and safety of mavacamten on a larger scale, across a variety of conditions.

COLLIGO-HCM, a global, retrospective data study, was presented at the European Society of Cardiology Congress 2025 in Madrid. The data indicated mavacamten’s association with reduced left ventricular outflow tract (LVOT) obstruction and improved symptom burden. The study also included a particularly racially diverse population (n = 278), including 23.2% Black, 5.4% Asian, and 4.3% Middle Eastern or North African patients.

A total of 59.9% of patients achieved ≥1 NYHA class improvement by week 24, and 86.5% of patients with ≥12 weeks of follow-up and 94.4% of those with ≥24 weeks of follow-up had an NYHA classification of II or below, including 30.9% who had an NYHA classification of I. These proportions increased consistently throughout week 96. Additionally, by week 36, 90.3% of patients achieved mean LVOT gradients of ≤30 mmHg at rest, and 76.8% after Valsalva provocation.

“COLLIGO challenges the one-size-fits-all approach, and it sort of broadens the scope of what should be second-line therapy and what should be first line,” Pankaj Arora, MD, said in an interview with 

. “Beta blockers and calcium channel blockers currently in the guidelines, they are first line, but when patients continue to have symptoms or elevated gradients, myosin innovation using mavacamten is being considered.”

During the follow-up period, mean left ventricular ejection fraction (LVEF) remained at or above 61% compared to a baseline value of 66%. Temporary interruption due to LVEF ≤50% occurred in 11 patients, and permanent discontinuation took place in 3 patients. New-onset atrial fibrillation was only recorded in 8 patients, which is consistent with previously reported data from other studies.

Most patients initiated mavacamten while also taking background medications, such as beta blockers or calcium channel blockers (n = 258). Of these, 26.4% discontinued ≥1 type of therapy, and 5% downtitrated their background medications after starting mavacamten. Arora highlighted this detail as a critical find, indicating potential room for further study.

Moreover, the nearly 60% of patients who improved ≥1 NYHA class highlight the potential for a paradigm shift in HCM treatment. Arora discussed what he views as an impending shift in the way clinicians and investigators will approach the disease and its effects.

“This is not just symptom management; it is functional restoration for these patients,” Arora told 

. “It’s time to recalibrate our scales, time to rethink what we define as successful therapy in obstructive HCM.”

Bristol Myers Squibb Presents Real-World Outcomes of Camzyos (mavacamten) Across Four Continents at the European Society of Cardiology (ESC) Congress 2025. Bristol Myers Squibb. August 29, 2025. Accessed September 15, 2025. 

https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Presents-Real-World-Outcomes-of-Camzyos-mavacamten-Across-Four-Continents-at-the-European-Society-of-Cardiology-ESC-Congress-2025/default.aspx

COLLIGO-HCM: A Multinational Observational Study of the Real-World Effectiveness of Mavacamten Among Patients with Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM) (COLLIGO-HCM). ClinicalTrials.gov identifier: NCT06372457. Updated April 18, 2024. Accessed September 15, 2025. 

https://clinicaltrials.gov/study/NCT06372457?term=COLLIGO-HCM&rank=1

Videos

Arora discusses the results of Bristol Myers Squibb’s observational study and the implications it has for the study of obstructive HCM and cardiology at large.

Off-label dose escalation of 2 interleukin (IL)-17A inhibitors, secukinumab and ixekizumab, may improve outcomes in certain cases among patients with 

, but new findings suggest the benefits should be weighed against costs and risks.

These findings and others regarding off-label adjustments to using ixekizumab and secukinumab among individuals living with psoriasis resulted from a recent letter to the editor authored in part by Jashin J. Wu, MD, from the Department of Dermatology at the University of Miami Miller School of Medicine.

Wu and coauthors highlighted that despite the well-known efficacy of IL-17A inhibitors in treating moderate-to-severe plaque psoriasis, many individuals need off-label dosing adjustments. Such shifts can include reduction, escalation, or interruption of medications.

“To evaluate the efficacy and safety of such regimens, a search of PubMed, Cochrane Library, Ovid MEDLINE and Embase was conducted for randomized controlled trials published between January 1, 2014 and December 6, 2024, assessing off-label dosing regimens in adults with moderate-to-severe psoriasis,” the investigative team wrote.

The investigators noted that the escalation of biologic use is sometimes considered by clinicians to improve one's response in psoriasis, but evidence suggests this strategy should be implemented selectively. For secukinumab, dose intensification from every 4 weeks (Q4W) to every 2 weeks (Q2W) is not routinely advised unless those on the medication weigh ≥90 kg.

Among those showing only partial improvement in their disease (PASI ≥75 but <90), a shift to Q2W led to a modest 7%–10% PASI-90 rate increase compared with Q4W, though this difference did not reach statistical significance. In additional data highlighted by the team, patients ≥90 kg saw more substantial benefits as a result of this strategy.

Escalation of one's ixekizumab dose during maintenance was shown in additional findings highlighted by Wu et al to yield greater advantages, with PASI-90 being observed in 79.5% of those on Q2W dosing versus 65.2% on Q4W (

< .001). Other data explored by the investigators suggested that dose de-escalation strategies, aimed at drug cost reduction or side effect limits, carry the risk of diminished effectiveness among patients.

Specifically, the research team pointed to findings showing that among secukinumab-treated individuals who had already reached PASI ≥90, maintenance at Q4W preserved higher PASI-90 rates (85.7%) as opposed to Q6W (74.9%) by the 52-week mark (OR 1.91, 95% CI 1.44–2.55). Similarly, the team pointed to findings showing that extending dosing to Q12W led to a 35% reduction in sPGA 0/1 rates at the 60-week mark relative to Q4W.

In pooled analyses from the UNCOVER trials, data also suggested that during induction, sPGA 0/1 responses were more robust with Q2W dosing compared to Q4W at the 12-week mark (81.8% versus 75%, 

< .001). Although some maintained adequate disease control with the diminished dosing, overall patterns suggested that less frequent administration of this drug would compromise outcomes. As such, Wu and coauthors highlighted the importance of individualizing such decisions.

The investigators pointed to additional data indicating that continuous therapy generally provides the most stable disease control, adding that despite this conclusion, treatment interruption does not always lead to irreversible loss of efficacy. IL-17A inhibitors are known to have a moderate relapse timeline, allowing certain patients to remain clear for months without treatment. Although not all individuals with psoriasis regain their initial level of improvement, many do, supporting the feasibility of temporary treatment pauses when clinically necessary. Additional data was noted as necessary to clarify whether reintroducing loading doses at the time of re-treatment improves outcomes, as study designs have differed.

Lastly, Wu and colleagues highlighted the central concern of safety with both escalation and de-escalation. Notably, the investigative team pointed to the consistent dose-dependent increase in serious adverse events that was observed with secukinumab and ixekizumab. However, they added that prior data suggest common mild infections with higher dosing. Anti-drug antibodies (ADA) are another concern. The team noted that this may be impacted by treatment interruption. In secukinumab retreatment-as-needed studies, ADA formation took place in 2 subjects on continuous therapy and a single patient on intermittent therapy, with neutralizing antibodies (nAbs) observed in 1 patient from each cohort. In the ixekizumab UNCOVER-1 and -2 extension trials, investigators had observed ADA in 19% of individuals on continuous treatment and 22.5% of those retreated after interruption, with nAbs present in 2 subjects from the re-treatment cohort.

“This study is limited by cross-trial comparisons, restricted generalizability due to selective trial populations and analysis of dose adjustments in patients meeting prespecified responses,” the team concluded.

 “Brodalumab and bimekizumab were excluded to maintain focus, as including all IL-17 inhibitors would significantly broaden the review and reduce the depth of analysis.”

Kim D, Babaei N, Cervantes M, Wu JJ. Off-label biologic regimens of IL-17A inhibitors for psoriasis. J Eur Acad Dermatol Venereol. 

Reich K, Puig L, Orsenigo R, et al. Secukinumab dosing optimization in patients with moderate-to-severe plaque psoriasis: results from the randomized, open-label OPTIMISE study. Br J Dermatol. 2020 Feb;182(2):304-315. 

. Epub 2019 Sep 8. PMID: 31102257. Accessed September 15, 2025.

Off-label dose escalation of IL-17A inhibitors, secukinumab and ixekizumab, may enhance outcomes for psoriasis patients, but the benefits must be balanced against potential costs and risks. Dose intensification, particularly for patients weighing ≥90 kg, can improve response rates, though not always significantly. Escalation of ixekizumab during maintenance showed notable advantages, while de-escalation strategies risk reduced effectiveness. Continuous therapy generally offers stable disease control, though temporary treatment pauses may be feasible. Safety concerns include dose-dependent adverse events and anti-drug antibodies, necessitating careful consideration of individualized treatment plans.

This letter explores off-label dosing adjustments for patients with psoriasis using the IL-17A inhibitors secukinumab and ixekizumab.

Dose Escalation of IL-17A Inhibitors for Psoriasis Effective, Though Risks Remain

On September 4, 2025, the US Food and Drug Administration (FDA) publicly released Lykos Therapeutics’ Complete Response Letter (CRL) on midomafetamine (MDMA) capsules for 

The FDA had initially issued a CRL on August 9, 2024, after its Psychopharmacologic Drugs Advisory Committee (PDAC) raised concerns on June 4, 2024, about safety, cardiovascular risks, and functional unblinding in trials.

 The committee noted that pivotal trials lacked systematic documentation of abuse-related adverse events, as required by the FDA, and provided insufficient data to guide clinicians on the duration of MDMA’s effect.

Other concerns included inconsistent therapy protocols, limited patient diversity, unclear separation of MDMA effects from psychotherapy, patient vulnerability due to acute impairment (i.e., impaired judgment), and inadequate therapist oversight.

The CRL, now public, lists 3 main concerns, 8 safety components, and 9 trial suggestions that do not affect approvability.

Failure to report positive or abuse potential adverse events.

The CRL noted that the study failed to collect important information deemed “positive” or “favorable” by participants, therapists, or investigators, and the FDA identified several unreported adverse events at multiple sites. Documenting such events is critical to assess abuse potential, as even seemingly positive experiences—like euphoria or mood changes—can signal risk.

The FDA also found inconsistencies between the MAPP2 protocol and the training/safety manual in defining adverse events. The manual excluded “positive or favorable effects,” while the protocol included any medical occurrence, whether related to the trial or not. The agency emphasized that all events relevant to abuse potential or impairment should have been reported.

2. Not evaluating MDMA’s durability beyond 18 weeks

The MAPP1 and MAPP2 trials did not test whether MDMA is durable beyond 18 weeks. Since PTSD is chronic, a durable benefit is required, especially given the proposed limited regimen of 3 treatment sessions.

The follow-up study (MPLONG) was inadequate due to design flaws: it had only 1 visit, variable timing (6 months–2 years), low enrollment with potential self-selection bias, and many participants received other therapies in the interim. As a result, the data do not establish whether MDMA’s benefits persist, if retreatment is needed, or how it should be managed long term—information essential for labeling and clinical use.

3. Selection bias with many participants having prior experience with MDMA

About 40% of participants had prior MDMA use, raising functional unblinding concerns. The FDA recommends a randomized, double-blind study excluding prior users, with pre-specified retreatment criteria, full adverse event reporting, and third-party data audit.

Detail any significant changes in the safety profile.

Present new safety data alongside original data, including adverse event frequencies for all indications and comparisons with prior reports.

Provide case report forms and narratives for participant deaths, discontinuations due to adverse events, and serious adverse events.

Highlight changes in common, less serious adverse events.

Update clinical study exposure information.

Summarize worldwide safety experience, including drug use in other countries.

Include English translations of current foreign labeling.

Other recommendations include routine labs (i.e. liver analytes, electrolytes) and vital signs, psychotherapy contribution (i.e. including an arm without psychotherapy), improving diversity, assessing MDMA-SSRI interactions, PK studies in hepatic/renal impairment, and lactation and pre-/post-natal studies.

“Lykos will continue negotiations with the FDA,” said MAPS Founder & President Rick Doblin in a statement.4 “MAPS will keep driving forward: incubating and accelerating new MDMA-focused research, training therapists…catalyzing humanitarian projects in high trauma/low resource areas of the world and building the infrastructure for a future where psychedelic-assisted healing and personal growth is not delayed by shifting politics but delivered as a matter of compassion and justice.”

Complete Response, NDA 215455. US Food and Drug Administration. August 8, 2024. Accessed September 15, 2025. 

https://psychedelicalpha.com/wp-content/uploads/2025/09/CRL_NDA215455_20240808.pdf

Derman C. FDA Rejects Application for MDMA-Assisted Therapy in PTSD, Calls for Additional Trial. 

. Published August 9, 2024. Accessed September 15, 2025. 

https://www.hcplive.com/view/fda-rejects-application-for-mdma-assisted-therapy-in-ptsd-calls-for-additional-trial

Kunzmann K. FDA Psychopharmacologic Advisory Committee Votes Against Supporting Effectiveness of MDMA for PTSD. HCP Live. Published June 4, 2024. 

https://www.hcplive.com/view/live-updates-fda-psychopharmacologic-advisory-committee-meeting-mdma-ptsd

MAPS Statement on FDA’s Public Release of Complete Response Letter for MDMA-assisted Therapy – Multidisciplinary Association for Psychedelic Studies – MAPS. Maps.org. Published September 5, 2025. Accessed September 15, 2025. 

https://maps.org/2025/09/04/fda-public-release-of-crl/

The FDA released a Complete Response Letter (CRL) for Lykos Therapeutics' MDMA capsules for PTSD, highlighting concerns about safety, trial design, and data reporting. Key issues include inadequate documentation of abuse-related adverse events, lack of data on MDMA's long-term efficacy, and selection bias due to prior MDMA use among participants. The CRL outlines three main concerns, eight safety components, and nine trial suggestions. Lykos plans to address these issues and continue negotiations with the FDA, while MAPS remains committed to advancing MDMA research and training therapists for future applications.

MDMA’s CRL, now available to the public, cites gaps in safety data, selection bias, and limited durability.

FDA Releases CRL Detailing Safety Concerns for MDMA-Assisted Therapy in PTSD

Rectal examinations are a routine yet often underutilized component of physical assessment across a wide range of clinical settings. While they are frequently associated with prostate evaluation, particularly in men, their scope is much broader and extends to the detection of multiple anorectal conditions.

Despite their importance, many clinicians report limited training or discomfort with performing rectal exams, which can contribute to underuse and misinterpretation of findings, some of the most common of which include hemorrhoids, anal fissures, perianal abscesses, fistulas, and skin tags. Beyond these, rectal exams can also reveal more serious conditions such as anal cancer or precancerous lesions related to human papillomavirus (HPV).

When performed properly, rectal exams remain one of the most valuable bedside tools for differentiating between common and clinically significant anorectal disorders, as highlighted at the 

2025 Gastroenterology & Hepatology Advanced Practice Providers (GHAPP) conference

 by Amy Stewart, NP, a GI Nurse Practitioner at Capital Digestive Care.

For further insight into her session at GHAPP and some of the key takeaways for NPs and PAs, the editorial team of 

 spoke with Stewart for the following Q&A:

: What are the goals of and take home messages from this session?

: My fun titled topic is "Everything Anal: What's Going on Back There?"

I think in school for many of us NPs and PAs were taught how to do a very basic rectal exam, which usually focuses on the prostate. But that's not our goal of a rectal exam in practice, right? So in practice, we need to distinguish between hemorrhoids, fissures, abscesses, fistula, HPV, and anal cancer without anyone ever really teaching us how to do this exam.

My talk is about all of the different kinds of common anal rectal conditions that you see and how to clinically examine for them, what you need specifically to do on your exam, what you're looking for. It also includes a lot of pictures so people can really see what they might see in clinic. The goal is for participants to really leave that talk feeling much more confident about a rectal exam, which is a really key piece of our job as NPs and PAs.

: What are the most common misconceptions or misdiagnoses you see when it comes to rectal exams?

: So I would say in general, less so from GI APPs, but certainly from my colleagues who work in urgent care and primary care, if you don't do a lot of rectal exams, it can be hard to understand the pathology of what you're looking for. I see many skin tags that are treated as active hemorrhoids, and you can put all the steroid cream you want on a skin tag, but it's not going to change or go away. So a component of my talk is the difference between the two.

Anal fissures are also often really commonly misdiagnosed or missed on exam. You can see them, but you really have to get into all the nooks and crannies of that perianal skin in order to see them. There's a lot of really good clinical pearls, if you will, in what you're looking for and what you're going to see on exam.

: Are there any other key takeaways you would like to highlight?

: I think just some other things that sometimes GIs don't recognize as often is HPV. So looking for condylomas or pre-cancerous changes from HPV, as they also specialize in anal HPV, but also herpes lesions are often misdiagnosed as fissures too. So I also have some information about that, and really kind of the clinical picture of how that presents for people as well.

Villanueva Herrero JA, Abdussalam A, Kasi A. Rectal Exam. StatPearls. February 18, 2023. Accessed September 15, 2025. 

https://www.ncbi.nlm.nih.gov/books/NBK537356/ 

Stewart reviews clinical pearls for performing rectal exams and explains common mistakes and misdiagnoses as well as how to avoid them.

Beyond the Prostate: Expanding the Role of the Rectal Exam With Amy Stewart, NP

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 September 5-September 13, 2025:

FDA Removes Sparsentan (Filspari) Advisory Committee Meeting for FSGS sNDA

The FDA determined no advisory committee is needed for sparsentan’s sNDA in FSGS, which remains under review with a January 2026 decision date.

Intercept Voluntarily Withdraws Obeticholic Acid (Ocaliva) for PBC From US Market

Intercept will withdraw obeticholic acid from the US PBC market after an FDA request and related clinical trial holds.

FDA Accepts FibroScan Letter of Intent for Surrogate Endpoint in MASH Trials

The FDA accepted a proposal to qualify FibroScan liver stiffness measurement as a surrogate endpoint for MASH trials, supported by multiple industry partners.

FDA Clears IND Application for EGEN-2784, a Genetically Engineered Pig Kidney, for ESKD

eGenesis received FDA clearance to begin clinical testing of a genetically engineered porcine kidney transplant in patients with ESKD.

First in Class Cat, Birch Allergen Blockers Succeed in Phase 3 Trials

Regeneron reported phase 3 success for novel allergen-blocking antibodies targeting cat and birch allergies, with confirmatory trials planned.

The recent healthcare updates include significant FDA decisions and clinical trial advancements. The FDA has decided against an advisory committee for sparsentan's sNDA in FSGS, with a decision expected in January 2026. Intercept is withdrawing obeticholic acid from the US PBC market following FDA requests. FibroScan's liver stiffness measurement is now a surrogate endpoint for MASH trials. eGenesis received FDA clearance for clinical testing of a genetically engineered pig kidney for ESKD. Regeneron reported successful phase 3 trials for allergen-blocking antibodies targeting cat and birch allergies.

An audio recap of the top 5 stories in healthcare news from the week of 9/5-9/13.

HCPLive 5 Stories in Under 5: Week of 09/07

Navigating New PBC Therapies and Treatment Monitoring With Ann Moore, NP

Primary biliary cholangitis (PBC) remains a challenging disease to manage, but advances in treatment are creating new opportunities to improve both disease outcomes and quality of life. While no therapy is perfect or universally effective, clinicians now have multiple options beyond first-line ursodeoxycholic acid (UDCA), making individualized care more achievable than ever.

, Ann Moore, NP, a nurse practitioner at Arizona Liver Health, broke down some of the latest advances in cholestatic disease and their impact on the field of hepatology.

In an interview with HCPLive, Moore emphasized the importance of monitoring and tailoring therapy based on the needs of the patient in front of you. With seladelpar (Livdelzi) and elafibranor (Iqirvo) now available as second-line treatment, she says providers now have options for how to individualize treatment.

“Years [went] by where we only had one option and there was no additional treatment,” Moore said. “Now we really have to individualize, and I think that's what's exciting about what's happening in treatment now.”

Despite expressing comfort with prescribing these newer agents, Moore noted long-term extension data are still needed, and questions remain about their impact on symptoms like pruritus and sleep.

She also underscores how far the field has come, noting that while PBC patients were once considered “rare” and left with limited options, today they are increasingly common in clinic, and management has shifted away from a “set it and forget it” model. With better tools for noninvasive monitoring, Moore says clinicians can more precisely track disease progression or improvement, adjusting therapy as needed.

“I think the key is recognizing inadequate response to first-line therapy and being aggressive with choosing your second-line therapy,” Moore said. “Don't wait a year. If a patient is not responding, if they're not where you want them to be, then consider second line therapy addition.”

Editors’ Note: This interview was recorded prior to Intercept Pharmaceuticals’ voluntary withdrawal of obeticholic acid (Ocaliva) from the US market on 

Brooks A. FDA Grants Accelerated Approval to Elafibranor (Iqirvo) for PBC. HCPLive. June 10, 2024. Accessed September 15, 2025. 

https://www.hcplive.com/view/fda-grants-accelerated-approval-to-elafibranor-iqirvo-for-pbc

Brooks A. FDA Grants Accelerated Approval to Seladelpar (Livdelzi) for Primary Biliary Cholangitis. HCPLive. August 14, 2024. Accessed September 15, 2025. 

https://www.hcplive.com/view/fda-grants-accelerated-approval-to-seladelpar-livdelzi-for-primary-biliary-cholangitis

Moore discusses the importance of individualizing PBC treatment now that multiple second-line therapies are available.

Epinephrine Nasal Spray Shows Comparable Efficacy to Injection, With Thomas Casale, MD

ARS Pharmaceuticals recently reported that 

epinephrine nasal spray (neffy) performed as effectively as epinephrine injections

 in treating anaphylaxis during routine clinical practice.

 Findings from this large-scale real-world analysis will be published later this year in 

Annals of Allergy, Asthma, and Immunology

, the official journal of the American College of Allergy, Asthma, and Immunology.

The company announced that the epinephrine nasal spray successfully treated about 9 out of 10 patients. A single 2 mg dose successfully treated 486 patients, with 59 patients requiring a second dose.

This finding was identical to the historic response rate found with epinephrine injection. Compared to the epinephrine injection’s 88.9% success rate, a single dose of epinephrine nasal spray resulted in an 89.2% success rate.

 spoke with investigator Thomas B. Casale, professor of Medicine and Pediatrics and chief of Clinical and Translational Research in the USF Health Morsani College of Medicine at the University of South Florida, about what this means for allergists.

“[The biggest benefit [of this study] is even though neffy, through a number of…trials to get FDA approval, show equivalent levels of epinephrine in the blood and relevant PD changes—that is, changes in pulse rate and blood pressure—a lot of physicians want to make sure: does it really work? And I think this study is very important in showing that it did,” Casale said. “Before this, there [were] only a couple of small clinical studies. One was 15 patients doing oral food challenges that showed that [epinephrine nasal spray] treated them successfully.”

The US Food and Drug Administration (FDA) approved epinephrine nasal spray, marketed as neffy, for type 1 allergic reactions in adults and children aged ≥ 4 years who weigh > 33 lbs. The approval opened a new anaphylaxis treatment for patients who fear needles or experience portability issues.

Many patients delayed or avoided auto-injectors due to their fears. In 2023, of the 20 million patients treated for severe reactions, only 3.2 million filled an auto-injector prescription, and half consistently carried it. Now, seen with this large analysis, epinephrine nasal spray not only eliminates many patients' fears but is as effective as having an injection.

“We know from a number of pieces of data that about 90% of patients get better with a single dose of epinephrine, and of the 10% that need a second dose, more than half of those get better with the second dose,” Casale said. “These data show that the numbers were very similar, and that with the first dose of neffy, 90% of patients got better when they had signs and symptoms of anaphylaxis. So again, very reassuring that have performed similar to what we've seen and noted with injectable epinephrine.”

Real-World Evidence Supports Clinical Effectiveness of neffy® (epinephrine nasal spray) in Patients Experiencing Anaphylaxis - ARS Pharmaceuticals. ARS Pharmaceuticals. Published 2025. Accessed September 12, 2025. 

https://ir.ars-pharma.com/news-releases/news-release-details/real-world-evidence-supports-clinical-effectiveness-neffyr

Derman, C. Epinephrine Nasal Spray as Effective as Injection for Anaphylaxis in Real-World Data. HCPLive. September 12, 2025. 

https://www.hcplive.com/view/epinephrine-nasal-spray-effective-as-injection-anaphylaxis-real-world-data

Real-world data show epinephrine nasal spray achieved an 89.2% success rate in treating anaphylaxis, nearly identical to the injection’s response rate.

Image Credit: US Food and Drug Administration

The US Food and Drug Administration (FDA) has approved bumetanide nasal spray (Enbumyst) for the treatment of edema associated with 

Announced by Corstasis Therapeutics on September 15, 2025, the FDA’s decision is in response to the unmet need in edema and fluid overload, which are the main causes of hospitalization and readmission for patients with CHF, cirrhosis, and CKD.

Loop diuretics are the first-line treatment for congestion, used to control volume status in patients with heart failure (HF). Most patients admitted to the hospital for HF are given intravenous (IV) loop diuretics irrespective of perfusion status. This is because the high plasma content in loop diuretics is needed to cross the threshold to rapid and effective diuresis. However, chronic use of loop diuretics can lead to a blunted response in acute decompensated HF, which is attributed to functional and structural tubular remodeling.

Bumetanide nasal spray is an intranasal loop diuretic designed for absorption through the nasal application. Developed to circumvent the absorption block presented by an overreliance on intravenous loop diuretics in managing diuresis, bumetanide nasal spray saw its star begin to rise with positive results from the RSQ-777-02 trial. It was conducted from December 2023 to April 2024 and included 68 adults, ages 18 to 55 years, without HF or risk of HF before enrollment.

"[Bumetanide nasal spray] was designed in direct collaboration with cardiologists to address practical challenges in the outpatient care setting," Brian Kolski, MD, chief medical director of Corstasis and director, non-invasive vascular lab at the Orange County Heart Institute and director of structural heart disease at Providence St. Joseph Hospital in Orange, California, said in a statement.

During the study, investigators gave patients all 3 forms of bumetanide – nasal, oral, and intravenous – in a varied order. The nasal spray was safe and well-tolerated throughout, with no significant nasal irritation observed. The team also noted an absorption variability of 27% in the nasal and IV forms of bumetanide, while the oral, pill form had a variability of >40%. Additionally, BNS had fewer treatment-emergent adverse events than the oral version.

Ultimately, bumetanide nasal spray met its primary endpoint of equivalence to oral bumetanide, and no significant difference in maximum plasma concentration was noted between the treatments. Additionally, a higher time to maximum concentration was observed in bumetanide nasal spray versus the oral tablets, indicating earlier detection of peak plasma drug concentrations after the nasal application.

Corstasis Therapeutics presented the results at the American Heart Association Scientific Sessions in 2024 and submitted the New Drug Application for BNS shortly afterwards. The FDA accepted the application in January of 2025.

"[Bumetanide nasal spray] offers the potential to change the standard of care by enabling earlier, outpatient intervention," Anuradha Lala-Trindade, MD, director of heart failure research at the Mount Sinai Fuster Hospital in New York, said in a statement. "This innovation may meaningfully improve outcomes while potentially easing the economic burden on the healthcare system."

According to the press release, Corstasis expects to launch bumetanide nasal spray in the US during the fourth quarter of 2025.

Corstasis Therapeutics. Corstasis Therapeutics Announces FDA Approval of ENBUMYST (bumetanide nasal spray) for the Treatment of Edema Associated with Congestive Heart Failure, Liver Disease, and Kidney Disease. 

. September 15, 2025. Accessed September 15, 2025. 

https://www.businesswire.com/news/home/20250915243998/en/Corstasis-Therapeutics-Announces-FDA-Approval-of-ENBUMYST-bumetanide-nasal-spray-for-the-Treatment-of-Edema-Associated-with-Congestive-Heart-Failure-Liver-Disease-and-Kidney-Disease

Biegus J, Ponikowska B, Canonico ME, Damman K, Palazzuoli A, Ambrosy AP. Bumetanide nasal spray: a novel approach to enhancing diuretic response and advancing ambulatory heart failure care?. Heart Fail Rev. 2025;30(5):1123-1126. 

American College of Cardiology. RSQ-777-02: New Nasal Spray Form of Bumetanide as Effective as Oral and IV Forms in Patients Without HF or HF Risk. November 18, 2024. Accessed September 11, 2025. 

https://www.acc.org/latest-in-cardiology/articles/2024/11/13/21/17/mon-930am-rsq77702-aha-2024

Iapoce C. FDA Accepts New Drug Application for Bumetanide Nasal Spray. 

. January 27, 2025. Accessed September 11, 2025. 

https://www.hcplive.com/view/fda-accepts-new-drug-application-for-bumetanide-nasal-spray

The FDA has approved bumetanide nasal spray (ENBUMYST) for treating edema linked to congestive heart failure, liver disease, and chronic kidney disease. This approval addresses the unmet need for effective outpatient management of fluid overload, a major cause of hospitalizations. Bumetanide nasal spray, developed to overcome limitations of intravenous loop diuretics, demonstrated safety and efficacy in clinical trials, showing equivalence to oral forms with fewer adverse events. The nasal spray offers potential for earlier intervention and improved patient outcomes, with a US launch expected in late 2025.

The US Food and Drug Administration has approved bumetanide nasal spray for the treatment of edema in outpatient heart, liver, and kidney diseases.

FDA Approves Bumetanide Nasal Spray for Edema with Congestive Heart Failure

The Medical Sisterhood: Calendars, Clinics, and Chaos, With Amy Chen, MD

In this episode of The Medical Sisterhood, host Mona Shahriari, MD, assistant clinical professor of dermatology at the Yale University School of Medicine and associate director of clinical trials at Central Connecticut Dermatology Research, is joined by her colleague and practice partner Amy Y. Chen, MD, a fellowship-trained dermatologist at Central Connecticut Dermatology and Middlesex Health. Together, the 2 physicians open up about the shared challenges of navigating demanding medical careers while raising young children, offering candid insights and practical strategies for clinician mothers.

Chen begins by describing the annual “September chaos,” when the back-to-school season collides with the unrelenting pace of clinical practice. With late school buses, shifting sports schedules, and unpredictable logistics, she stresses the importance of flexibility and organization. Her solution has been to centralize all family activities in a color-coded Google Calendar—managed solely by her—to reduce confusion, alongside weekly index cards posted at home outlining pickup and drop-off duties. These small systems, she explains, help her family maintain order amid the flux of school and extracurricular commitments.

The conversation turns to the “mental load” of motherhood, a burden that often defaults to women despite the presence of supportive partners. Chen candidly admits that she frequently wakes in the night worrying about forgotten school supplies or missed deadlines. Shahriari likens this experience to having dozens of browser tabs open at once—some frozen, some endlessly buffering—yet all demanding attention. Both acknowledge that this invisible labor can lead to burnout, underscoring the need for physician mothers to carve out moments of rest and transition.

Chen shares that she finds small windows of “me time” in unexpected places: sitting quietly in her car after work, scrolling social media before bed, or simply allowing herself to stare at the wall during her children’s practices. Shahriari echoes the importance of these mental resets, describing how she switches deliberately between her “dermatologist hat” and her “mom hat” to remain present in each role.

On parenting philosophy, Chen advises relinquishing the pursuit of perfection—accepting mismatched outfits, less-than-Pinterest-worthy lunches, or occasional scheduling mishaps as inevitable trade-offs in a busy life. For her, the non-negotiables remain high-quality patient care and being present for her children’s important moments.

Both physicians reflect on how motherhood has deepened their empathy for patients, particularly those juggling similar demands. They close by reminding listeners that self-compassion and grace are essential: even for high-achieving women in medicine, it is both normal and necessary to let some things slide.

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This episode highlights the challenges of navigating demanding medical careers while raising young children, with insights and practical strategies.

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