New Insight with Dr. Veeral Sheth

Diabetes Dialogue

Don’t Miss a Beat

Crisis Point

 and joined the MJH Life Sciences team in April 2021. He graduated from The College of New Jersey with a degree in Journalism and Professional Writing. He enjoys listening to records, going to concerts, and playing with his cat Squish. You can reach him at 

Antonio Risitano, MD, PHD
Credit: Aplastic Anemia and MDS International Foundation

Topline data from the Phase 3b APPULSE-PNH trial reported the benefit of twice daily oral monotherapy iptacopan (Fabhalta) for adult patients with 

paroxysmal nocturnal hemoglobinuria (PNH)

Announced by Novartis on December 6, 2024, the 24-week treatment results revealed an improvement in the average hemoglobin (Hb) level versus baseline with iptacopan. Safety outcomes remained consistent with previously reported data, according to the company.

“These new results add to the body of evidence reinforcing that [iptacopan] can benefit both patients previously treated with anti-C5 therapies studied in the APPULSE-PNH and APPLY-PNH trials and complement-inhibitor naive patients studied in the APPOINT-PNH trial,” lead trial investigator Antonio Risitano, MD, PHD, Chair of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit, Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria at the AORN San Giuseppe Moscati, said in a statement.

A rare, chronic, and serious complement-mediated blood disorder, PNH is estimated to affect nearly 10 to 20 million people globally.

 People with PNH exhibit an acquired mutation in hematopoietic stem cells leading to the production of red blood cells susceptible to premature destruction by the complement system and ultimately, intravascular and extravascular hemolysis.

Iptacopan was developed as an oral, Factor B inhibitor of the alternative complement pathway. The drug was granted US Food and Drug Administration (FDA) approval in December 2023 for treating adults with PNH.

 In August 2024, iptacopan was granted accelerated approval by the FDA for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN), with ongoing development in multiple other complement-mediated diseases.

APPULSE-PNH is a Phase 3b multicenter, single-arm, open-label study evaluating the efficacy and safety of twice-daily oral iptacopan 200 mg in adults with PNH switched from anti-C5 therapies, including eculizumab or ravulizumab. A total of 52 participants received iptacopan for 24 weeks.

Enrolled participants were required to be on a stable regimen with anti-C5 therapies for ≥6 months before screening, with average Hb levels of ≥10 g/dL and no red blood cell transfusions during this period. A primary endpoint was the change from baseline Hb levels after 24-week treatment with iptacopan.

Upon analysis, after 24 weeks of treatment with Fabhalta, the average Hb level improved compared with baseline. The safety profile of iptacopan monotherapy was consistent with previously reported data.

“Treatment goals for patients with PNH have greatly evolved, and we can now aim to resolve signs and symptoms of disease in most patients,” Risitano added.

 “It is promising to see this evolution, and we will continue to make progress to best support these patients.”

Adding to the growing positive body of evidence for iptacopan, Novartis has announced these clinically meaningful data from APPULSE-PNH will be presented at an upcoming 2025 medical meeting.

“These data reinforce our confidence in [iptacopan], the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful hemoglobin improvement, regardless of previous treatment experience,” David Soergel, MD, global head of the cardiovascular, renal and metabolism development unit at Novartis, said in a statement.

New phase IIIB data shows Novartis Fabhalta® improved hemoglobin levels in adult patients with paroxysmal nocturnal hemoglobinuria who switched from anti-C5 therapy. Novartis. December 6, 2024. Accessed December 6, 2024. https://www.novartis.com/news/media-releases/new-phase-iiib-data-shows-novartis-fabhalta-improved-hemoglobin-levels-adult-patients-paroxysmal-nocturnal-hemoglobinuria-who-switched-from-anti-c5-therapy.

Shah N, Bhatt H. Paroxysmal Nocturnal Hemoglobinuria. [Updated 2023 Jul 31]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK562292/

Abigail Brooks M. FDA approves Iptacopan (Fabhalta) for paroxysmal nocturnal hemoglobinuria. HCP Live. December 6, 2023. Accessed December 6, 2024. https://www.hcplive.com/view/fda-approves-iptacopan-fabhalta-for-paroxysmal-nocturnal-hemoglobinuria.

Campbell P. IPTACOPAN receives accelerated approval for reducing proteinuria in IGA nephropathy. HCP Live. August 8, 2024. Accessed December 6, 2024. https://www.hcplive.com/view/iptacopan-receives-accelerated-approval-for-reducing-proteinuria-in-iga-nephropathy.

Articles

Topline results from APPULSE-PNH show the positive efficacy and safety of twice-daily oral iptacopan in adults with PNH switched from anti-C5 therapies.

News

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

Clinical

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

Hematology

Anemia

Iptacopan Boosts Hemoglobin Levels in Adults with PNH in Phase 3b Trial

Andreas Kremer, MD, PhD, MHBA, is the head of hepatology at University Hospital Zurich in Switzerland.

Seladelpar’s Impact on Pruritus

 treatment landscape has long been limited to just a handful of agents with limited efficacy, 2024 saw a major shift in PBC management with the addition of 2 new second-line therapies.

Seladelpar (Livdelzi), a proliferator-activated receptor (PPAR) delta agonist, was one of these recent additions to the PBC treatment armamentarium and was a major topic of discussion at 

The Liver Meeting 2024 from the American Association for the Study of Liver Diseases (AASLD)

Along with a late-breaking presentation highlighting seladelpar’s sustained efficacy and long-term safety in a 2.5-year interim analysis of the ongoing open-label phase 3 ASSURE study, another abstract presentation focused on the agent’s impact on pruritus in the RESPONSE trial.

In this segment, which is the final installment of a 4-part series, Andreas Kremer, MD, PhD, MHBA, head of hepatology at University Hospital Zurich, explains seladelpar’s impact on pruritus symptoms in patients with PBC.

Kremer delves into the promising outcomes of recent studies on seladelpar and its potential to alleviate pruritus in PBC, specifically focusing on a posthoc analysis revealing that patients with moderate to severe pruritus who were treated with seladelpar achieved a significant reduction on the numeric rating scale (NRS) for symptom severity.

In addition to improvements in pruritus among patients who reported itch at baseline, Kremer pointed out patients who reported no pruritus at baseline who were treated with seladelpar did not report any new-onset pruritus, whereas 25% of those on placebo developed pruritus during the same period. In totality, the findings underscore the potential antipruritic efficacy of seladelpar alongside its primary anti-cholestatic and anti-inflammatory properties.

Kremer points to an exciting future for PBC management, with new therapies on the horizon poised to improve both disease outcomes and quality of life for patients.

Editors’ note: Kremer has relevant disclosures with AbbVie, AstraZeneca, Bayer, CymaBay, Gilead, GlaxoSmithKline, Intercept, Mirum, Takeda, Ipsen, and others.

Brooks A. FDA Grants Accelerated Approval to Seladelpar (Livdelzi) for Primary Biliary Cholangitis. 

. August 14, 2024. Accessed December 6, 2024. 

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

Brooks A. Seladelpar (Livdelzi) Demonstrates Long-Term Benefit for Primary Biliary Cholangitis. 

. November 15, 2024. Accessed December 6, 2024. 

https://www.hcplive.com/view/seladelpar-livdelzi-demonstrates-long-term-benefit-primary-biliary-cholangitis

Videos

The treatment landscape for primary biliary cholangitis (PBC) has evolved with the introduction of new second-line therapies, notably seladelpar, a PPAR delta agonist. Recent studies, including the ASSURE and RESPONSE trials, highlight seladelpar's sustained efficacy, long-term safety, and significant impact on pruritus symptoms in PBC patients. Notably, seladelpar reduced pruritus severity in patients with moderate to severe symptoms and prevented new-onset pruritus in those without baseline symptoms. These findings suggest seladelpar's potential to enhance disease management and patient quality of life.

Andreas Kremer, MD, PhD, MHBA, explains seladelpar’s impact on pruritus in PBC based on findings from a posthoc analysis of the RESPONSE trial.

Hepatology

Cholangitis

cholangitis page, resources on the topics of medical news and expert insight into cholangitis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on research, treatment, and drug development in PBC, PSC, and more!

Emerging Second-Line Therapies in PBC

In this segment, which is part 3 of a 4-part series, Andreas Kremer, MD, PhD, MHBA, head of hepatology at University Hospital Zurich, reviews emerging second-line therapies in PBC.

Kremer provides an overview of promising advancements in the treatment of pruritus in PBC, focusing on 2 recently FDA-approved PPAR agonist therapies: elafibranor and seladelpar. He explains how these drugs target PPAR to address the underlying mechanisms of PBC. Both seladelpar, a specific PPAR delta agonist, and elafibranor, a dual PPAR alpha and delta agonist, exhibit anticholestatic and anti-inflammatory properties, making them effective options for treating the disease's root causes.

Highlighting recent clinical advancements, Kremer highlights seladelpar's performance in a phase 3 trial where the drug met its pre-specified secondary endpoint for significantly reducing pruritus severity, as measured by the numeric rating scale. He additionally called attention to a posthoc analysis from the study that revealed a strong anti-pruritic effect in certain patient subgroups, offering additional hope for tailored treatment approaches.

The treatment landscape for primary biliary cholangitis (PBC) has evolved significantly with the introduction of two new second-line therapies in 2024, including seladelpar, a PPAR delta agonist. Seladelpar's efficacy and safety were highlighted at The Liver Meeting 2024, with studies showing its impact on pruritus and long-term benefits. Andreas Kremer, MD, PhD, discussed the advancements in PBC treatment, focusing on seladelpar and elafibranor, both PPAR agonists with anticholestatic and anti-inflammatory properties. Seladelpar's phase 3 trial results showed significant pruritus reduction, offering hope for personalized treatment strategies.

Andreas Kremer, MD, PhD, MHBA, reviews emerging therapies in PBC, focusing specifically on seladelpar and its impact on pruritus.

Unmet Needs for Pruritus Management in PBC

In this segment, which is part 2 of a 4-part series, Andreas Kremer, MD, PhD, MHBA, head of hepatology at University Hospital Zurich, explains current unmet needs for pruritus care in PBC management.

Kremer calls attention to a critical gap in managing pruritus in patients with PBC, highlighting the unmet need for therapies that address both the underlying disease and symptom burden. He notes that current in-label treatments, such as ursodeoxycholic acid and obeticholic acid, have not demonstrated efficacy in alleviating pruritus, and in some cases, these therapies may even exacerbate the symptom despite their proven ability to reduce cholestasis.

Kremer stresses the importance of developing new, in-label drugs that are not only effective but also safe and well-tolerated for long-term use. He expresses optimism about ongoing advancements, noting promising developments in the pharmaceutical pipeline targeting rare liver diseases that offer hope for more comprehensive treatment strategies that address both the clinical and symptom-related aspects of PBC.

The treatment landscape for primary biliary cholangitis (PBC) has evolved with the introduction of new second-line therapies, including seladelpar, a PPAR delta agonist. Recent studies presented at The Liver Meeting 2024 highlighted seladelpar's sustained efficacy and safety, as well as its impact on pruritus. Current treatments like ursodeoxycholic acid and obeticholic acid have limited efficacy in managing pruritus, underscoring the need for new therapies. Ongoing advancements in the pharmaceutical pipeline offer hope for more comprehensive treatment strategies addressing both clinical and symptom-related aspects of PBC.

Andreas Kremer, MD, PhD, MHBA, explains shortcomings of traditional PBC therapies for addressing pruritus and what advantages newer agents may offer.

Understanding Pruritus in Primary Biliary Cholangitis

In this segment, which is part 1 of a 4-part series, Andreas Kremer, MD, PhD, MHBA, head of hepatology at University Hospital Zurich, explains the burden of pruritus in PBC and its negative impact on affected patients’ quality of life.

Kremer highlights the widespread prevalence of pruritus in PBC, affecting anywhere between 30% and 80% of patients depending on the source. Beyond its severity measured on the numeric rating scale, Kremer describes how pruritus profoundly impacts quality of life, disrupts sleep, induces fatigue and depression, and impairs daily functioning and work capacity.

He goes on to describe the psychological challenges it poses, particularly in social and professional settings, where visible discomfort can exacerbate emotional strain. Ultimately, the multifaceted toll pruritus takes on patients underscores the critical need for effective management strategies.

The treatment landscape for primary biliary cholangitis (PBC) has evolved with the introduction of new second-line therapies, including seladelpar, a PPAR delta agonist. Seladelpar's efficacy and safety were highlighted at The Liver Meeting 2024, with studies showing its impact on pruritus, a common and debilitating symptom of PBC. Pruritus affects 30-80% of PBC patients, significantly impairing quality of life, sleep, and daily functioning. The psychological and social challenges it poses emphasize the need for effective management strategies to alleviate this burden on patients.

Andreas Kremer, MD, PhD, MHBA, describes the burden of pruritus in PBC and the toll it can take on patients’ quality of life.

Expert dermatologists discuss the role of IL-17 A/F inhibition in the management of hidradenitis suppurativa. 

Hidradenitis Suppurativa

Hidradenitis Suppurativa 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 dermatology research, treatment, and drug development.

Dermatology

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

FDA News

Understanding, Leveraging IL-17 A/F Inhibition with Bimekizumab in HS

In this 5-part, expert-led series, a pair of dermatologists break down how the FDA approval of bimekizumab impacts the treatment landscape of HS.

Examining Treatment Landscape, Options in Hidradenitis Suppurativa

Tim Smith joined the MJH Life Sciences team as an assistant editor for HCPLive in August 2022. He graduated from UC Berkeley with a degree in political science, working in multimedia journalism as a staff writer prior to joining MJH. In his spare time, he enjoys reading, watching TV, listening to podcasts, and rock climbing. You can contact him at 

These data highlight some of the most notable findings related to airline travel issues among those with food allergies or with family members with such allergies.

Allergy

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

Food Allergy

Study Highlights Global Data on Food Allergy-Related Airline Travel Concerns 

Chelsie Derman is an assistant editor for 

who covers psychiatry, sleep medicine, and geriatrics. 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 new study showed patients with MASLD have fragmented nocturnal sleep with frequent awakenings per night and a session on healthy sleep habits did not improve sleep parameters.

Sleep

On the HCPLive sleep page, resources on the topics of medical news and expert insight into sleep medicine can be found. Content includes articles, interviews, videos, podcasts, and breaking news on sleep apnea, insomnia, narcolepsy, and more.

MASH / MASLD

The HCPLive MASH & MASLD page is a comprehensive resource for clinical news and insights on metabolic dysfunction-associated steatotic liver disease and steatohepatitis. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for fatty liver disease, steatohepatitis, and more.

Study Suggests Fragmented Nocturnal Sleep May Play a Role in MASLD Development

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

Phase 1/2 data prompted the RMAT designation for HLHS shortly after Mesoblast announced positive phase 3 data for HFrEF. 

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.

Cell Therapy Gets RMAT Designation for HLHS, Improves Survival in Heart Failure With Reduced Ejection Fraction 

Jörn Schattenberg, MD, is a professor of medicine and director of the Metabolic Liver Research Program at the University Medical Center Mainz in Germany.

Schattenberg explains pegozafermin’s mechanism of action and describes the importance of preventing progression to cirrhotic MASH.

Conference

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

Jörn Schattenberg, MD: Pegozafermin and Preventing Progression to Cirrhosis in MASH

Laura Ross, PA-C, CLS, AACC, is a physician assistant and clinical lipid specialist in cardiology at the Park Nicollet Heart and Vascular Center in St. Louis Park, MN. She is a member of ACC's Interventional Leadership Council and the Prevention Work Group, and she is president of the Academy of Physician Associates in Cardiology.

Greg Duck, PA-C shared insights from his CAPP 2024 presentation on tailoring medical management and effective treatment of methamphetamine-induced cardiomyopathy.

Structural Heart Disease

The APAC Recap

Too many scientific congresses or meetings and not enough time? Welcome to the APAC Recap! 

Co-hosted by the Academy of Physician Associates in Cardiology (APAC) and HCPLive, this multimedia series will bring you clinically relevant insights and perspectives from APAC members as they return from the dozens and dozens of meetings the group attends throughout the year. 

The APAC Recap: Cardiomyopathy at CAPP Live 2024 with Greg Duck, PA-C

Abigail Brooks is an assistant 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.

This month in review spotlights GI pipeline updates and news from the 2024 NASPGHAN and ACG annual meetings.

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.

Irritable Bowel Syndrome

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

Ulcerative Colitis

Ulcerative Colitis condition center page, resources on the topics of medical news and expert insight into colitis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on UC research, treatment, and drug development.

Gastroenterology Month in Review: November 2024

The phase 3 OLYMPIA 1 study highlights the benefits among those with prurigo nodularis of targeting interleukin-31 signaling using nemolizumab.

Study Finds Nemolizumab Significantly Improved Signs, Symptoms in Prurigo Nodularis

Perspective from neurologists on the current and future applications for artificial intelligence across a bevy of neurologic conditions. 

Hospital Medicine

The HCPLive hospital medicine page is a comprehensive resource for clinical news and insights on the emergency medicine specialty. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for emergent care, and more.

Neurology

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

Latest News

Iptacopan Boosts Hemoglobin Levels in Adults with PNH in Phase 3b Trial

Seladelpar’s Impact on Pruritus

Emerging Second-Line Therapies in PBC

Unmet Needs for Pruritus Management in PBC

Understanding Pruritus in Primary Biliary Cholangitis