 JR Humphreys

 saw the addition of 2 new second-line treatment options for 

 with the FDA accelerated approvals of elafibranor (Iqirvo) and seladelpar (Livdelzi).

A chronic and progressive autoimmune liver disease affecting intrahepatic bile ducts, PBC treatment has historically relied on ursodeoxycholic acid. While this continues to be the only first-line therapy, many patients do not respond to or are unable to tolerate it, underscoring the need for more treatment options to prevent progression to cirrhosis and eventual liver failure as well as to improve symptoms most negatively impacting patients, including pruritus and fatigue.

For additional insight into the clinical impact of having elafibranor and seladelpar as treatment options for PBC and what’s next in PBC research, the editorial team of 

 spoke with Aparna Goel, MD, an associate professor of medicine at Stanford University and a transplant hepatologist in the division of gastroenterology and hepatology at Stanford University Medical Center.

 Clinically, what has been the impact of now having these new second-line therapies as options to offer patients?

: It's great to have options for our patients. When we have 3 different approved second-line therapies that we can offer our patients who are not responding to Ursodial as well as we want them to, it's all about going through the historical data and looking at what long-term data we have for these drugs, what the efficacy rates are, what the side effect profile is, and understanding what other medications they are on that might impact that which drug you try to use, if they have concomitant itch or not, if they have significant fatigue or not.

It's very nuanced in terms of which medicine you might recommend, but I think it's important for us to review all these medications with our patients. Our patients are excited, I think many patients love seeing their alkaline phosphatase levels reach normal values. For the first time in decades, some of them are getting to normal values with the addition of second-line therapy that they can tolerate.

Beyond the biochemical response that patients are seeing with these drugs, I think both of them have also shown potential benefits in terms of improvements in pruritus and improvements in fatigue. It's really nice to be able to see that impact for our patients in terms of their quality of life as well.

 Are there any drugs or trials in PBC that you're most looking forward to seeing more of in 2025?

: PBC has definitely been saturated with new second-line therapies. There’s actually another PPAR currently being studied in PBC, saroglitazar, which is a dual PPAR-alpha/gamma agonist, so we'll have to see the results of that.

In terms of PBC, I think what is exciting to see on the horizon is what the long-term benefits of these medications are going to be. It's not just about improving alkaline phosphatase levels, but are we confident that the improvement in the alkaline phosphatase levels is the equivalent of an improvement in liver-related health? Are we preventing transplantations? Are we improving rates of hepatic decompensation? Only with time will we truly be able to determine that.

We also need to understand more about the side effect profile of these medications. There was an increased risk of fractures for both seladelpar and elafibranor in the studies, so I think really understanding what the PPARs are doing to the bones will be interesting.

We need to better understand these drugs’ impact on quality of life. These are exploratory endpoints that were studied in both of the trials, and while there were signals for improved itch and improved fatigue, I think we have to understand what the mechanism is there and how we can potentially use these medicines to help patients with these symptoms, even if their alkaline phosphatase levels might not be incredibly abnormal.

There's a big push in PBC of not just treating to mild elevations of alkaline phosphatase, but really pushing to normal values. I said 40-60% of the population of those with PBC are nonresponders, but that's if we look at an alkaline phosphatase threshold of 1.5 or 1.67 times up the limit of normal. If we look at anyone that's above normal after a year of Ursodial, that number probably goes up to closer to 70%, maybe even 75%. I think there's a big population of patients who will benefit from bringing their alkaline phosphatase levels down to normal, and it’s important to understand the clinical impact of that and what the benefit is for our patients.

Editors’ note: Goel has no relevant disclosures.

Brooks A. FDA Grants Accelerated Approval to Elafibranor (Iqirvo) for PBC. HCPLive. June 10, 2024. Accessed March 24, 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 March 24, 2025. 

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

In 2024, the FDA approved two new second-line treatments for primary biliary cholangitis (PBC): elafibranor and seladelpar. These options are crucial for patients who do not respond to or cannot tolerate ursodeoxycholic acid, the only first-line therapy. The new treatments have shown potential in improving symptoms like pruritus and fatigue, enhancing patients' quality of life. Future research will focus on long-term benefits, side effects, and the impact on liver health. Saroglitazar, another PPAR agonist, is currently under investigation for PBC.

Goel describes the clinical impact of having 2 newly approved second-line PBC therapies and looks ahead to what’s next and where more research is needed.

Understanding Elafibranor and Seladelpar for PBC, with Aparna Goel, MD

Aparna Goel, MD

Through nearly 3 decades as a healthcare leader, I have never experienced anything like joining 

 as chief executive officer this past January. I stepped in to lead Orange County, California’s only licensed chemical dependency recovery hospital, one of just a handful in the state, in the midst of a double pandemic—the opioid crisis and COVID-19. More than 10 million Americans 

 prescription opioids in the last year; over half a million people have died of COVID-19. My experience was tested by these intertwined crises, requiring adaptable, caring leadership that puts people first.

 in substance abuse and an increase in overdoses. As with healthcare providers the world over, COVID-19 has transformed the way we care for patients. Staff must observe strict protocols for protection, distancing, and patient admission to keep people and environment safe. These infection prevention measures are standard practice in a variety of healthcare settings but can have a 

 on our vulnerable patients, who often come to us in a high state of anxiety. 

The patients, seeking support for addiction, must be tested by nasal swab for COVID-19 and subsequently placed in isolation for between 24 and 48 hours—which can feel like an eternity for someone struggling with a substance abuse disorder. The facility, in accordance with local health restrictions, has prohibited family and friends from visiting since last March; loved ones provide a critical lifeline and source of support for patients recovering from addiction. Pandemic restrictions were keeping patients safe from one disease, even as they created obstacles to surviving another.

Every patient we see brings their own psychosocial, economic, mental health, even legal difficulties into treatment. We help them navigate the personal crises, such as a breakup or job loss, that may be entangled with their addiction. Adding to that the 

uncertainty, fear, and loss of a global pandemic

 has exacerbated the anxiety and stress staff and patients experience every day. That is why I make sure to start the workday with a meeting to discuss the status and needs of every patient, to deliver truly empathetic, customized care. 

When each workday brings the unexpected, maintaining calm is vital. My primary responsibility is encouraging and supporting the team providing care to our patients. That means being present, being engaged, and getting involved—not hunkering down in an office. Some days I feel like I am the orchestra conductor. Not being clinical myself I do not play the instrument, but I know how and when it needs to be played. This is what I love about leadership.

That emphasis on mindfulness extends to the care we provide our patients, too. People struggling with addiction can become trapped in their own heads, consumed by their personal situation. Our approach, particularly with new evening activities programming like ping pong and charades, is to enable them, even if briefly, to focus on something beyond the problems that got them here. Enjoying laughter, companionship, play, sunshine, and a good meal can help ground us, reminding us of the basic joy of the human experience.

Finding joy is truly important when dealing with addiction, a disease laden with secrecy and shame. The fight to break down the stigma surrounding addiction is constant, whether it be with insurance carriers, demanding they provide our patients comprehensive care, or the shame carried by patients themselves. We work hard to change mindsets, so that patients understand that they are not some grim statistic, but rather a person with worth and dignity who is battling a disease like any other.

The need for more mental health resources in addiction treatment facilities is becoming ever more acute: Treatment centers are seeing a new, more deadly class of drugs emerge: Fentanyl is a powerful synthetic opioid 

 than morphine. Illegal use of this prescription drug is killing young people across the country. 

There are promising new treatments: Cutting edge medications, such as Suboxone, Sublocade, and Vivitrol that allow those struggling with addiction to avoid relapse and lessen the pain of withdrawal. But even with increasingly advanced tools, we must shine a light on this tragic epidemic. 

Helping those facing the searing personal struggles of addiction is tough, unpredictable work, and can take an emotional toll. Leaders must set boundaries to avoid burnout. Now, I understand how self-care—evening walks with my husband and dog, time with friends and family, plenty of sleep, and good nutrition—help me do my job better. Working in a high stress environment such as addiction treatment, you absolutely must practice what you preach regarding coping strategies. An agile healthcare leader, like an orchestra conductor helping each player’s notes soar, is tuned in, able to offer the guidance to help employees and patients thrive.

Barbara Kennedy, MHA, FACHE, is Chief Executive Officer, Laguna Treatment Hospital, American Addiction Centers.

The views expressed in this piece reflect those of the author, not necessarily those of the publication.

Clinicians and experts interested in responding to this piece, or submitting their own articles to

Stress induced by COVID-19 is driving opioid addiction risks. An expert weighs in on addressing one matter to resolve another.

Leading Through Twin Healthcare Crises

Barbara Kennedy, MHA, FACHE

HCPLive Five – Allergy Updates at AAAAI 2025

 2025 Academy of Allergy, Asthma, & Immunology (AAAAI) 

annual meeting in San Diego to connect with colleagues, explore late-breaking data, and attend hundreds of sessions.

 Allergy Team reported live from San Diego, conducting dozens of interviews and covering late-breaking research. In this iteration of the HCPLive Five, the team highlighted standout video interviews, covering topics such as insect allergy testing, chronic rhinosinusitis, omalizumab for food allergies, oral immunotherapy with autoclaved peanuts, and the WAYPOINT phase 3 trial assessing tezepelumab for nasal polyp management.

When to Test for Insect Sting Allergies, with David Golden, MD

David Golden, MD, from Johns Hopkins University, discussed the pitfalls of over-testing for insect sting allergies. He emphasized that a positive test does not confirm an allergy, as many people with positive results experience no reaction to stings.

Testing should be reserved for patients with anaphylactic reactions, as unnecessary testing can lead to undue worry and even disqualify individuals from opportunities like military service. Unlike peanut allergies, insect allergy tests do not predict reaction severity, making it crucial for clinicians to avoid misinterpreting strong test results as indicators of severe allergy risk.

When an Allergist Should Refer Endoscopic Sinus Surgery for Chronic Rhinosinusitis, with Kent Lam, MD

Kent Lam, MD, from Eastern Virginia Medical School at Old Dominion University, discussed when allergists should refer patients with chronic rhinosinusitis for endoscopic sinus surgery; surgery is recommended when symptoms persist despite treatment with topical steroids, saline irrigation, or antibiotics.

The procedure aims to improve sinus drainage and ventilation rather than remove tissue. Lam emphasized setting patient expectations regarding risks such as bleeding, scarring, and symptom persistence.

Omalizumab Performs Better Than Oral Immunotherapy for Food Allergy, with Robert Wood, MD

Robert Wood, MD, from Johns Hopkins Medicine, discussed the OUtMATCH trial comparing omalizumab and oral immunotherapy (OIT) for food allergies. The study found omalizumab superior in the intent-to-treat analysis (36% vs 19%;

 = .031), but per-protocol analysis—excluding dropouts—showed similar effectiveness (

OIT had a high dropout rate due to side effects, while omalizumab was well tolerated. Wood emphasized the need for individualized treatment, as OIT may offer long-term immune changes, but omalizumab provides a safer alternative with fewer side effects.

Patients Tolerate Oral Immunotherapy Better with Autoclaved vs Blanched Peanuts, with Casey Cohen, PhD

A study found that peanut-allergic patients tolerated oral immunotherapy better with autoclaved peanuts than with blanched peanuts. Casey Cohen, PhD, from McGill University, explained that autoclaving—using high temperature and pressure—reduces peanut-specific immunoglobulin binding, resulting in fewer severe reactions.

In a double-blind trial of 10 participants, the median tolerated dose was significantly higher for autoclaved peanuts (300 mg vs. 9 mg). No participants required epinephrine for autoclaved peanuts, whereas all were for blanched peanuts.

Tezepelumab Lowers Nasal Polyp Severity in CRSwNP During Phase 3 WAYPOINT, with Joseph Han, MD

The phase 3 WAYPOINT trial demonstrated that tezepelumab significantly reduced nasal polyp severity in patients with severe chronic rhinosinusitis with nasal polyps (CRSwNP), lowering the need for surgery by 92%. Tezepelumab significantly improved nasal congestion, sense of smell, and overall symptom scores at 52 weeks.

Investigator Joseph Han, MD, from Eastern Virginia Medical School, highlighted the near elimination of surgery in the treatment group. These findings support tezepelumab as a promising treatment option for CRSwNP, reducing reliance on systemic steroids and surgical intervention.

GlaxoSmithKline, Regeneron Pharmaceuticals, and AstraZeneca Pharmaceuticals for Lam, Genentech USA for Wood, none for Cohen, and GlaxoSmithKline, Genzyme Corporation, Regeneron Healthcare Solutions, Medtronic, Merck Sharp & Dohme, Optinose, AstraZeneca Pharmaceuticals, Aerin Medical, and PFIZER for Han.

Counting down the most impactful news at major medical meetings, it’s the HCPLive Five! This episode focuses on 5 key allergy updates from the 2025 American Academy of Allergy, Asthma, and Immunology (AAAAI) annual meeting.


David Golden, MD

Dermatologists Drs Tina Bhutani and Scott Boswell and patient Kanya Oul share resources and tools available to cope with generalized pustular psoriasis (GPP).

Tips for Managing Generalized Pustular Psoriasis (GPP)

This is a video synopsis/summary of a panel discussion involving Tina Bhutani, MD; J. Scott Boswell, MD; and a patient.

In the concluding segment of the discussion, the patient emphasizes the importance of healthcare providers listening to their patients and believing their experiences. She credits her doctor with saving her life and underscores the significance of finding a reliable healthcare provider who understands the patient's condition. The patient encourages providers to educate themselves and be proactive in referring patients for appropriate care.

The speaker echoes the importance of education, stressing the need for healthcare professionals to stay updated on advancements in their field. He emphasizes the role of primary care physicians and emergency medicine practitioners in recognizing and treating severe skin conditions like generalized pustular psoriasis (GPP). The speaker shares his proactive approach to adopting new treatments for the benefit of his patients.

Overall, the discussion highlights the importance of patient-provider communication, education, and collaboration in effectively managing conditions like GPP. The participants express gratitude for the opportunity to share their insights, underscoring the value of such discussions in improving patient care.

Video synopsis is AI-generated and reviewed by HCPLive editorial staff.

Dermatologists Drs Tina Bhutani and Scott Boswell and a patient share resources and tools available to cope with generalized pustular psoriasis (GPP).

J. Scott Boswell, MD 

Before we transition out here, any thoughts on the future pipeline, phase 2 data, or phase 1 data, what’s the future here?

There are lots of potential options, more second-generation FMT [fecal microbiota transplantation] products that we can talk about, but we also have other narrow-spectrum antibiotics in the pipeline. The closest is ridinilazole, which is similar to fidaxomicin, and it’s going to target 

 and kill it very well, but also spare our commensal microbiota. The phase 2 data look great, it helped prevent recurrence among patients who received ridinilazole compared to oral vancomycin. That’s exciting to me.

There are 2 other microbiome-based therapies that have finished phase 2 clinical trials and 1 that has finished a phase 1 clinical trial. Going alphabetically, CP101 is a full-spectrum microbiome-based therapy that recently finished a phase 2 clinical trial and showed a 74.5% versus close to 60% success rates. This is going to go to a phase 3 clinical trial in the near future. There’s another one that is a room temperature stable or nonfrozen formulation of a capsule called RBX7455, and that one finished a phase 1 study with a 90% success rate, and a larger trial is being planned at this time. Then finally, moving on to what I call designer microbiome-based therapies, where the field is going to go, is VE303, which is from a donor-independent bacterial consortia. Recently in a press release, they had their phase 2 data presented, where the high-dose VE303 was statistically significant and clinically meaningfully different from the placebo in preventing recurrent 

 infection. There is lots of excitement going on in today’s world.

There’s one more. There’s a company now with a particular single strain of bacterium that thrives on a particular oligosaccharide that’s found in certain kinds of fibers where they’re hoping that this 1 strain, which would be a true probiotic, would be able to reduce the risk for recurrences of 

 We’ve really painted the entire spectrum of all the different therapeutic options. We start with full-spectrum stool, which is basically the entire biodiversity. We move toward another company that basically focuses on the different consortia, or the ecological niches, where defects in metabolism can then just be plugged right back in. Then we have these companies that are more designer that have found out that maybe we can pick this handful of species alone and give them back to our patients. And then companies that are probably more savvy have figured out that there may be some species that have multiple functions, and you may just need to give 1 species back. And of course, as we are scientists, we can figure out what we don’t need to give an organism back, we can just find the products that they produce or the proteins that are on their surface and use those. There’s a whole different spectrum of therapeutic options, which is great for our patients because at that point, we have options to choose what’s going to work best for patients in different scenarios.

So the future is bright. I would like to ask all of our participants to give us a final thought.

One of the things that we sometimes neglect is how devastating this disease is to our patients. We talked about the diarrhea itself, we talked about the hospitalization, the epidemiology, the impact on hospitals, but at the same time, there are a number of patients who have their lives turned upside down because of the first infection or the recurrences. I’ve taken care of patients who have lost their jobs, I’ve taken care of patients who can’t be with their families, who ask me if they can see their grandchildren. I’ve seen patients who have developed this post-traumatic stress disorder from having 

 infections. If we think about the thousands and thousands of recurrences that we see each year, there are thousands and thousands of people who are not the same after this infection. I’d like to stress to everyone who’s watching to read these guidelines and look to see what is the appropriate way to treat these patients the first time because that gives them the best chance of success of beating this infection so that they can go back to their normal lives afterward.

That was beautifully said, Carl. This infection creates a huge burden on individual patients, but also on our health care system. I’d also like to mention that the microbiome field and microbiome therapeutics are burgeoning right now. We are learning new things every single day, and some of these microbiome-targeted therapies are ultimately going to be studied in other diseases too, so hopefully, we can make an impact elsewhere in addition to 

 infections. And the last thing I’ll say is, eat your fiber and close the toilet lid when you flush.

I’ll add 2 things. One thing to add to Carl’s point that is paramount is educating our patients on the impact of this illness. When you see a patient for the first time, don’t just hand out an antibiotic prescription and say, “You’ll be fine.” Patient education is paramount. There are resources that are available at the CDC’s [Centers for Disease Control] website, NIH [National Institutes of Health] has resources, and also the C Diff Foundation is a tremendous resource that I suggest to my patients. The other point that I would make is that as these microbiome-based therapies become mainstay, they’re going to move from tertiary care centers, from infectious disease and GI [gastrointestinal] doctors to empower primary care providers, family practice providers, nurse practitioners, physician assistants, who are the first touchpoints that our patients sometimes have with recurrent 

 They should start thinking that they should be and will be able to prescribe these therapies to our patients.

. If you enjoyed the content, please subscribe to our e-newsletters to receive upcoming 

 and other great content right in your inbox.

Closing out their discussion on Clostridioides difficile infection, panelists share closing thoughts on novel pipeline therapies.

Looking Toward the Future of CDI Management

James A. McKinnell, MD

 Let’s shift to the other stuff coming in the future. There are other mechanisms being investigated in therapeutics in atopic dermatitis. I’ll start with topicals.

There’s a new topical JAK inhibitor that has been studied in ages 12 and older. It’s probably as strong or a little stronger than triamcinolone 0.1% as a steroid, but it’s a nonsteroid. There’s no atrophy. It may have more potency than our other nonsteroidals without a lot of irritancy, burning, or stinging at all. We’re really excited about that.

Tapinarof is a drug in a totally different class of medicines. It’s an aryl hydrocarbon receptor modulator. This is being developed both for psoriasis and atopic dermatitis. 

Another PDE4 inhibitor is under study as well. Another topical JAK inhibitor, delgocitinib, is being studied in both chronic hand eczema and atopic dermatitis. They’re not ready to be written yet, but these are other medicines that will give us different topical options.

Peter, do you want to talk about biologics?

Absolutely. We have some new biologic agents that are in development. In particular, we have 2 new IL-13 inhibitors that are in late-stage development, so that’s going to be exciting. It seems they’ll be similar to dupilumab but a different molecule. It may be for patients who have had an adverse event with dupilumab or if it didn’t work fully. This may be another option. This is going to be nice. It seems like they’re going to have a similar safety profile and lack of lab monitoring. That’s exciting.

We have an IL-31 inhibitor that’s also in development. That’s interesting because some people call IL-31 the master itch cytokine. That would be neat because that could answer our question we asked at the beginning. Is this the itch that rashes? If so, how much? Of course, nothing is quite that pure. IL-31 is doing other things too, but the early data seem very promising, so that’s exciting to me as well.

 There are some oral JAK inhibitors coming down the pike as well. They are well along the way. Baricitinib and upadacitinib have been approved for other indications and are being studied in phase 3 trials for atopic dermatitis. There’s also a new molecule, abrocitinib. These will be different. They’ll be oral. There are some more issues with monitoring blood work, but that will give another potential approach to some of our patients. Two of the 3 JAKs have been studied in ages 12 and older, and the other is being studied in adults but going younger. There’s lots of new stuff happening. 

This has been an incredible discussion and very practical, reflecting on all the changes that are happening in the field. Let’s get final thoughts from each of our participants. Jeffrey? 

I’ll go back to education. Educate the pediatrician and make sure the pediatrician educates the family and takes the time. This is not an easy process. This is not an easy disease state. If we do a good job outlining to the families what they need to do, we can see much better outcomes. That goes without saying for everything that primary care doctors do. This forum is going to be great for people to take a look and hear what’s coming on and where we should be.

We all have to work together in a therapeutic alliance. We need to get the patients on board. We need all of the specialists and primary care practitioners to work together because we’re all together for the same concept. We want the patient and their family to be better. When we bring all our forces together, it’s so much more powerful. There’s great synergy.

These are exciting times in the world of atopic dermatitis. The world is going to be a better place with all these new therapies on the way and with the advent of Dupixent 4 years ago. It’s a good time to have atopic dermatitis. We can do many more things to help.

Thank you. Great contributions from everyone today. Thank you for watching this HCPLive® Peer Exchange. If you enjoyed the content, please subscribe to our e-newsletters to receive upcoming Peer Exchanges and other great content right in your in-box. Thank you.

Lawrence F. Eichenfield, MD; Jeffrey M. Bienstock, MD, FAAP; Peter A. Lio, MD; and Elizabeth A. Swanson, MD, review additional mechanisms being investigated in the pipeline for the treatment of atopic dermatitis. 

Emerging Agents for the Treatment of Atopic Dermatitis 

Lawrence F. Eichenfield, MD, Rady Children’s Hospital-San Diego 

Experts discuss strategies for making dry eye care an appealing practice option for optometry students and supporting their success in this specialized field.

Novel Treatments and Diagnostics for Dry Eye

Novel Treatment and Diagnostics for Dry Eye

Marjan Farid, MD  

Crinecerfont Shows Lasting Efficacy in Treating Adult CAH, with Richard Auchus, MD, PhD

According to a presentation by Richard Auchus, MD, PhD, professor of endocrinology and internal medicine at the University of Michigan Health, 1-year data from the phase 3 CAHtalyst adult trial displays the efficacy of crinecerfont (CRENESSITY) in treating patients with 

 to discuss the team’s findings and the possible future implementation of crinecerfont on a broad scale.

 in December 2024 to treat CAH in conjunction with glucocorticoids, crinecerfont functions by reducing excessive production of adrenal androgen, which reduces the amount of glucocorticoid treatment necessary to return patients to equilibrium.

The 24-week, double-blind, placebo-controlled trial compared patients with classic CAH on supraphysiologic glucocorticoid (GC) doses who were randomized in a 2:1 ratio to either crinecerfont or placebo. This was followed by a 24-week open-label period in which all participants received crinecerfont. GC doses were kept stable for the first 4 weeks of both periods and decreased as tolerated toward more physiologic levels while maintaining or improving androstenedione relative to baseline.

A total of 182 adult patients were included, ranging from 18-58 years of age. Patients receiving consistent crinecerfont saw a -27.3% change in glucocorticoid doses, compared to -10.3% in the placebo group (least-squares mean difference, -17 percentage points; 

 <.001). At week 4, androstenedione levels decreased with crinecerfont (-299 ng/dL) but increased with placebo (45.5 ng/dL) (least-squares mean difference, -345 ng/dL; 

The presented data concerned the second 24-week period, examining the placebo patients as they were crossed over to crinecerfont. Auchus explained these outcomes, highlighting similar reductions in glucocorticoid dosing.

“The clinical pearl from this is that they had their glucocorticoid dose reduced once a month, much more slowly than every 2 weeks that we reduced it in the crinecerfront group,” Auchus told 

. “I think the important point is that when using the drug clinically, you should add the crinecerfont, you should wait until the labs are under control, and then you should very slowly taper the glucocorticoid dose as you would, for example, in someone with Cushing’s disease that has just been cured with surgery.”

Auchus also noted a significantly lower number of side effects in the crossed-over cohort than the crinecerfont cohort, largely those associated with glucocorticoid withdrawal such as headaches and fatigue.

Discussing alternative measurement methods, Auchus highlighted increases in bone turnover markers, noting small but significant improvements in bone density.

“Both turnover markers went up, interestingly, in both the placebo group and the crinecerfont group, but more in the latter, who had their glucocorticoid dose lowered further,” Auchus said. “There were some small but not statistically significant improvements in bone density at year one. So, I think that is a positive effect on bones, or at least a reduction of the negative effect on bones that the glucocorticoid therapy is giving these people.”

Auchus ultimately provided advice for clinicians, indicating the importance of patience and calling for more endocrinologists need to provide care for patients with CAH.

“A lot of adult endocrinologists don’t like taking care of this disease because it’s very hard: you either know that you’re doing damage to these patients by giving them a lot of glucocorticoid, or you know that they’re reducing the glucocorticoid to something physiological, but you’re letting their androgens rise and you say that there’s nothing we can do about that,” Auchus said. “My hope is that this makes it easy for any adult endocrinologist to transition a patient from the pediatric endocrinologist in good control with or without crinecerfont and then manage them for many decades, without a lot of angst and worry about concomitant comorbidities.”

Editors' Note: Auchus reports disclosures with Adrenas Therapeutics, Corcept Therapeutics, Neurocrine Biosciences, Novo Nordisk, Recordati Rare Diseases, Sparrow Pharmaceuticals, and others.

US Food and Drug Administration. FDA Approves New Treatment for Congenital AdrenalHyperplasia. December 13, 2024. Accessed July 21, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-congenital-adrenal-hyperplasia

Neurocrine Biosciences. Neurocrine Biosciences Presents One-Year Data Showing Sustained Efficacy of CRENESSITY (crinecerfont) in Adult Patients, at ENDO 2025. PR Newswire. July 14, 2025. Accessed July 21, 2025. 

https://www.prnewswire.com/news-releases/neurocrine-biosciences-presents-one-year-data-showing-sustained-efficacy-of-crenessity-crinecerfont-in-adult-patients-at-endo-2025-302503650.html

Auchus RJ, Hamidi O, Pivonello R, et al. Phase 3 trial of Crinecerfont in adult congenital adrenal hyperplasia. New England Journal of Medicine. 2024;391(6):504-514. doi:10.1056/nejmoa2404656

Presented at ENDO 2025, crinecerfont exhibited sustained androgen reduction when combined with glucocorticoids.

Richard Auchus, MD, PhD

Chambliss also discussed other research she is working on looking to understand effects on children with asthma.

Understanding Effects of Pollution Sources in Neighborhoods, with Sarah Chambliss, PhD

New research into pollution sources and asthma complications has found that semiconductor fabrication plants (SFPs) are located disproportionately in census tracts with higher indicators of social vulnerability and a greater proportion of Black and Latinx residents and, slightly increasing population-based incidence of asthma-related emergency department visits.

These findings were presented by Sarah Chambliss, PhD, postdoctoral fellow at University of Texas at Austin, at the 

2025 American Academy of Allergy, Asthma, & Immunology (AAAAI)

 annual meeting in San Diego from February 28 – March 3.

The investigators found that there was around a 1% increase in asthma-related emergency department visits per unit of distance from the SFPS after adjusting for other socioeconomic factors, which, while small, adds up over time and with close proximity.

“Honestly, if people know that their kids are at higher risk of having worse asthma, and they just make a big deal out of it, they can prevent these factories from going into their neighborhoods, like that's why these factories don't exist in neighborhoods with more resources, because people complain about it. So, it's really as long as you have the advocacy, you can [do something about it],” Chambliss said.

 spoke with Chambliss to learn more about her research and other efforts she is engaged in to try and address issues with pollution sources in neighborhoods. She also stressed the importance of advocacy for these neighborhoods and discussed other research she is conducting to better understand the effects of pollution sources on children with asthma.

“We can get much more specific with the effects of air pollution on asthma morbidity like asthma exacerbations or symptom days, if we look at individuals instead of populations. So I'm currently working with a couple of groups that have cohorts of children, where they've measured, in some cases, their symptom load and their asthma control levels, and then in other cases, they've looked at them over time, and they've looked at their lung function growth. With both of those cohorts, we'll be able to really tease out these social factors much better from air pollution, because we'll know specifically what these each person is experiencing,” Chambliss said.

Chambliss's research is supported by an NIH grant.

Chambliss S, Zigler C, Warner A, et al. Understanding the Role of Pollution Sources in Intracity Racial and Ethnic Health Disparities: Proximity to Semiconductor Facilities, Neighborhood Demographic Characteristics, and Asthma-related Emergency Department Visits. Presented at: AAAAI/WAO Joint Congress, San Diego; 2/28-3/3. Abstract 865

Authors