Alex Hillenbrand is an assistant editor for 

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

 news in the third quarter of 2026 included 5 

, and nephrotic syndrome, along with 2-year trial data for therapies first approved on the basis of proteinuria.

IgAN accounted for much of the quarter's regulatory activity. Atacicept (Trutakna) received accelerated approval in July, and final ORIGIN 3 data in September showed it preserved kidney function through 2 years. Iptacopan (Fabhalta) converted to full approval on 2-year data, and 2-year VISIONARY results for sibeprenlimab (Voyxact) will support an application for traditional approval.

In CKD, finerenone (Kerendia) gained an indication for patients with 

, while FIND-CKD data extended its evidence to nondiabetic CKD. Obinutuzumab (Gazyva) was approved for childhood-onset idiopathic nephrotic syndrome and remains under priority review in primary membranous nephropathy.

Coverage also focused on detection and access. A joint European Society of Cardiology (ESC) and European Renal Association (ERA) guideline recommended CKD screening for every patient newly diagnosed with cardiovascular disease, the American Kidney Fund highlighted how often CKD goes undiagnosed, and new research found that fewer than half of patients referred for kidney transplant begin evaluation.

Catch up on the quarter's nephrology coverage from the HCPLive editorial team below.

FDA Approves Atacicept (Trutakna) for IgA Nephropathy

On July 7, 2026, the FDA granted accelerated approval to atacicept-vymj (Trutakna), a dual B-cell activating factor (BAFF) and a proliferation-inducing ligand (APRIL) inhibitor, to reduce proteinuria in adults with primary IgAN at risk of progression. The decision was based on an interim ORIGIN 3 analysis showing a 42% reduction in proteinuria versus placebo at 36 weeks.

Final ORIGIN 3 data reported in September showed atacicept reduced the risk of kidney disease progression by 76% compared with placebo in 428 patients. Annualized estimated glomerular filtration rate (eGFR) decline through 104 weeks was 0.6 mL/min/1.73 m² per year with atacicept and 5.6 with placebo. Vera Therapeutics plans to submit for full approval in the fourth quarter of 2026.

Atacicept Cuts IgAN Progression Risk 76% in ORIGIN 3 Final Data

FDA Grants Full Approval to Iptacopan (Fabhalta) in IgAN

On July 17, 2026, the FDA converted the accelerated approval of Novartis' iptacopan (Fabhalta) to traditional approval to slow kidney function decline in adults with primary IgAN at risk of progression.

The decision was supported by 2-year data from the phase 3 APPLAUSE-IgAN trial, in which iptacopan significantly slowed eGFR decline compared with placebo.

FDA Approves Furosemide Injection (Furoscix ReadyFlow) for Edema in HF, CKD

On July 24, 2026, the FDA approved MannKind's Furoscix ReadyFlow, an autoinjector that delivers subcutaneous furosemide, for edema in adults with heart failure (HF) or CKD.

The autoinjector cuts administration time from 5 hours with the existing on-body infusor to under 10 seconds, giving patients an at-home option for fluid overload.

FDA Approves Finerenone (Kerendia) for CKD and Type 1 Diabetes

On September 16, 2026, the FDA approved finerenone (Kerendia) to reduce urinary albumin-to-creatinine ratio (UACR) in adults with CKD associated with type 1 diabetes.

In the phase 3 FINE-ONE trial, finerenone reduced UACR by 28% compared with placebo at month 6. Hyperkalemia occurred in 10.1% of patients receiving finerenone and 3.3% receiving placebo.

FDA Approves Obinutuzumab (Gazyva) for Idiopathic Nephrotic Syndrome

FDA Grants Obinutuzumab Priority Review as Primary Membranous Nephropathy's Potential First Therapy

On September 25, 2026, the FDA approved obinutuzumab (Gazyva) for relapse prevention in patients aged 2 years and older with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome in complete remission. In the phase 3 INShore trial, 95.5% of patients receiving obinutuzumab maintained complete remission at week 52, compared with 73.2% receiving mycophenolate mofetil.

Earlier in the quarter, on July 14, 2026, the FDA granted priority review to obinutuzumab in primary membranous nephropathy. In the phase 3 MAJESTY trial, 36.9% of patients achieved complete remission at 2 years versus 5.7% with tacrolimus. A decision is expected by November 2026.

Kidney Compass: VISIONARY 2-Year eGFR Data in IgA Nephropathy

On August 3, 2026, Otsuka announced 2-year results from the phase 3 VISIONARY trial showing that sibeprenlimab (Voyxact), an anti-APRIL antibody, returned the annualized rate of kidney function decline in IgAN to a near-physiologic level.

On Kidney Compass, Dana Rizk, MD, of the University of Alabama at Birmingham, reported an annualized eGFR slope of +0.3 mL/min/1.73 m² per year with sibeprenlimab versus a decline of 4.2 with placebo. Sibeprenlimab received accelerated approval in November 2025, and the eGFR data will support a supplemental application for traditional approval.

Sibeprenlimab (Voyxact) Stabilizes Kidney Function in IgAN

Kidney Compass: FIND-CKD Extends Finerenone's Benefit to Non-Diabetic CKD

The phase 3 FIND-CKD trial found that finerenone slowed eGFR decline and reduced cardiorenal risk by 23% in nondiabetic CKD over 32 months. Rajiv Agarwal, MD, MS, discussed how the results extend finerenone's evidence beyond diabetic kidney disease.

On Kidney Compass, Brendon Neuen, MBBS, PhD, reviewed a prespecified glomerulonephritis subgroup analysis showing consistent benefit across IgAN, focal segmental glomerulosclerosis, and membranous nephropathy.

FIND-CKD Expands Finerenone Evidence Beyond Diabetic CKD, With Rajiv Agarwal, MD

New ESC, ERA Guideline Calls for Universal CKD Screening in Cardiovascular Disease

The ESC and ERA published their first joint guideline on cardiovascular disease and CKD, recommending screening with both eGFR and UACR for all patients with newly diagnosed cardiovascular disease.

The guideline also recommends statin-based therapy regardless of lipid levels and expanded use of sodium-glucose cotransporter 2 (SGLT2) inhibitors, finerenone, and semaglutide in CKD.

Nearly 9 in 10 US Adults With CKD Don't Know They Have It, With Pranav Garimella

Pranav Garimella, MBBS, MPH, chief medical officer of the American Kidney Fund, discusses why 87% of US adults with CKD are unaware of their diagnosis and what the US Preventive Services Task Force (USPSTF) should consider in its evidence review on CKD screening.

He covers gaps in eGFR and UACR testing among patients with type 2 diabetes, high-risk groups for screening, and the need for repeat testing to confirm CKD.

Why Fewer Than Half of Referred Patients Reach Kidney Transplant Evaluation

NYU Langone investigators used electronic health record data from 1850 hospitals to track 720,348 patients referred for kidney transplant. Of those, 48% began evaluation, 19% were waitlisted, and 10% underwent transplant.

Patients with severe obesity, those in rural areas, and those treated at small centers or programs in the South were less likely to progress. Allan B. Massie, PhD, encouraged nephrologists to follow up with patients after referral.

Articles

Nephrology in Q3 2026: FDA approvals for atacicept, iptacopan, finerenone, and obinutuzumab, plus 2-year IgAN data and CKD screening.

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Chronic Kidney Disease

10 Nephrology Headlines You Missed in Q3 2026

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for 

. He is also the executive producer of two podcasts: 

. Previously, he was the producer of the Lungcast podcast. He graduated from UC Berkeley and worked in multimedia journalism as a staff writer prior to joining MJH. You can contact him at tsmith@mjhlifesciences.com.

 saw a run of regulatory decisions and comparative efficacy data across inflammatory, autoimmune, and oncologic skin disease. 

 approvals expanded treatment options for dermatomyositis, adolescent alopecia areata, and superficial basal cell carcinoma. New phase 3 and indirect comparison data, meanwhile, sharpened the picture within the oral psoriasis treatment class.

Among the most significant Q3 developments was the FDA approval of brepocitinib (Lisraya), the first oral therapy indicated for adults with dermatomyositis. The agency also approved baricitinib (Olumiant) for adolescents with severe alopecia areata and Ameluz photodynamic therapy for superficial basal cell carcinoma. In clinical research, head-to-head phase 3 data comparing zasocitinib with deucravacitinib were presented at the 

European Academy of Dermatology and Venereology (EADV) Congress

. Pfizer also reported topline phase 3 results for ritlecitinib in nonsegmental vitiligo, and a new matching-adjusted indirect comparison weighed risankizumab against icotrokinra in plaque psoriasis.

The following review of the third quarter of 2026 highlights 6 key pieces of the 

FDA Approves First Oral Treatment of Dermatomyositis in Adult Patients

In one of the most consequential regulatory decisions of Q3 2026, the FDA approved brepocitinib (Lisraya) tablets for adults with dermatomyositis on August 27. Developed by Priovant Therapeutics, brepocitinib is a once-daily oral inhibitor of tyrosine kinase 2 (TYK2) and Janus kinase 1 (JAK1). The decision makes it the first oral drug indicated for the disease.

The approval was based on a randomized, double-blind, placebo-controlled phase 3 trial of 241 adults who received brepocitinib 30 mg, brepocitinib 15 mg, or placebo once daily. At Week 52, patients on the 30-mg dose achieved a higher mean Total Improvement Score (TIS) versus placebo and were more likely to reduce corticosteroid use by Week 48. The 15-mg dose did not significantly improve the primary endpoint.

"For too long, patients with dermatomyositis have faced a significant unmet need for effective treatments, often relying on therapies meant for other diseases," Nikolay Nikolov, MD, director of the Office of Immunology and Inflammation in the FDA Center for Drug Evaluation and Research, said in a statement.

FDA Approves Baricitinib for Adolescents With Severe Alopecia Areata

On September 25, the FDA approved Eli Lilly's baricitinib (Olumiant) for patients aged 12 years and older with severe alopecia areata, expanding the oral JAK inhibitor's 2022 adult indication. The decision was supported by 36-week results from the adolescent cohort of the phase 3 BRAVE-AA-PEDS study. Participants in this cohort were aged 12–17 years and had a median baseline Severity of Alopecia Tool (SALT) score of 100.

At Week 36, 42% of patients on baricitinib 4 mg and 27% on 2 mg achieved a SALT score of 20 or less, compared with 5% on placebo. Rates of SALT 10 or less reached 37% and 21%, respectively, versus 2% with placebo. Lilly reported a safety profile in adolescents consistent with prior adult data, and the drug retains the boxed warning common to JAK inhibitors.

"The evidence supporting Olumiant's approval is particularly meaningful because it comes from a Phase 3 study specifically designed for pediatric patients, helping clinicians and families make more informed decisions when considering advanced therapies," said Brittany Craiglow, MD, adjunct associate professor of dermatology at Yale School of Medicine.

Ameluz Treatment Becomes First FDA-Approved PDT for Skin Cancer

In a September 14 decision, the FDA approved Biofrontera's supplemental New Drug Application (sNDA) for Ameluz. Ameluz is aminolevulinic acid hydrochloride topical gel, 10%, used with the BF-RhodoLED lamp series, and the new indication covers superficial basal cell carcinoma (sBCC) in adults. The approval makes Ameluz the first photodynamic therapy (PDT) regimen approved by the agency for a skin cancer. It is also now the only topical PDT in the US indicated for both actinic keratosis and a skin malignancy.

The decision was supported by a randomized, double-blind, vehicle-controlled phase 3 trial of 187 adults with histologically confirmed sBCC. The composite primary endpoint was clinical and histological complete response at 12 weeks after the final PDT cycle. It was met by 66% (95/145) of patients on Ameluz PDT versus 5% (2/42) on placebo-PDT. Biofrontera plans to launch the sBCC indication between late Q4 2026 and Q1 2027.

"This approval reflects years of disciplined investment in the clinical development of Ameluz, and the latest stage in the ongoing expansion of our PDT platform," Hermann Luebbert, chief executive officer and chairman of Biofrontera, said in a statement.

EADV 2026: Zasocitinib Tops Deucravacitinib for PASI 100 in Plaque Psoriasis

Following topline results reported in Q2, full data from the phase 3 LATITUDE Atlas trial (NCT06973291) were presented at EADV Congress 2026 in Vienna, Austria. The trial enrolled 606 adults with moderate-to-severe plaque psoriasis. At Week 16, 36.5% of patients receiving zasocitinib 30 mg once daily achieved PASI 100, versus 13.9% receiving deucravacitinib 6 mg once daily (

Zasocitinib also outperformed deucravacitinib on all key secondary endpoints. These included PASI 90 (62.5% vs 34.0%) and a static Physician Global Assessment (sPGA) score of 0 (43.2% vs 18.5%). The FDA accepted Takeda's New Drug Application for zasocitinib under priority review in September 2026, and the European Medicines Agency is also reviewing the agent.

"The data from the Phase 3 trials provide a more complete picture of once-daily oral zasocitinib, demonstrating rapid, durable and consistent skin clearance alongside meaningful improvements in itch and quality of life," said Linda Stein Gold, MD, director of dermatology clinical research at Henry Ford Health in Detroit and principal investigator for LATITUDE Atlas.

Ritlecitinib Improves Repigmentation in Nonsegmental Vitiligo Phase 3 Data

On July 30, Pfizer announced positive topline results from the phase 3 TRANQUILLO and TRANQUILLO 2 trials of ritlecitinib (Litfulo) in nonsegmental vitiligo (NSV). Ritlecitinib is a once-daily oral inhibitor of JAK3 and TEC family kinases. Combined, the trials enrolled 2,174 patients with active or stable NSV across 271 sites worldwide.

Both trials met their US co-primary endpoints at Week 52. Significantly more patients on ritlecitinib than on placebo achieved at least 75% improvement in the Facial Vitiligo Area Scoring Index (F-VASI75) and at least 50% improvement in the Total Vitiligo Area Scoring Index (T-VASI50). Exact response rates were not disclosed in the topline release, and Pfizer reported no new safety signals. Based on these results, the company plans to pursue global regulatory filings for ritlecitinib in adults with NSV.

"In these trials, ritlecitinib demonstrated significant improvements in both facial and total body repigmentation, reinforcing its potential to help support a new treatment paradigm with systemic therapies for patients whose disease burden warrants more than localized treatment," said Iltefat Hamzavi, MD, senior staff physician in the department of dermatology at Henry Ford Health.

Risankizumab Versus Icotrokinra: New Data on Adults With Moderate-to-Severe Psoriasis

In July, a matching-adjusted indirect comparison (MAIC) published in Dermatology and Therapy evaluated risankizumab against icotrokinra in adults with moderate-to-severe plaque psoriasis. Icotrokinra is an oral targeted peptide blocking the IL-23 receptor. The investigators, including Linda Stein Gold, MD, of Henry Ford Health, matched individual patient data from the UltIMMa-1, UltIMMa-2, and IMMhance trials to published aggregate results from the ICONIC-LEAD, ICONIC-ADVANCE-1, and ICONIC-ADVANCE-2 trials.

At Week 16, risankizumab showed significantly greater placebo-adjusted response rates across all assessed endpoints. These included PASI 90 (71.4% vs 49.9%; 

 < .001). Differences emerged by Weeks 4 to 8 and persisted through Week 52 in unanchored analyses, although the investigators noted indirect comparisons cannot replace randomized head-to-head trials.

"In the absence of head-to-head trials, this study uses matching-adjusted indirect comparison (MAIC) to compare [risankizumab] with [icotrokinra] in the treatment of adult patients with moderate-to-severe psoriasis," Stein Gold and coauthors wrote.

Editor’s note: This summary has been edited for grammar and clarity using artificial intelligence tools.

Q3 2026 dermatology recap: FDA approvals in dermatomyositis, adolescent alopecia areata, and sBCC, plus new psoriasis and vitiligo findings.

Plaque Psoriasis

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

Q3 2026 Recap: Dermatology News & Updates

Linda Stein Gold, MD, is a dermatologist at Henry Ford Health. 

Zasocitinib achieved PASI 100 in 36.5% of patients versus 13.9% with deucravacitinib at Week 16.

Stein Gold attributed the gap to differences in TYK2 selectivity and duration of IL-23 blockade.

She now considers next-generation oral TYK2 inhibitors a first-line option after topical therapy.

Zasocitinib, an investigational next-generation tyrosine kinase 2 (TYK2) inhibitor, may allow clinicians to aim for completely clear skin with an oral agent for 

, according to Linda Stein Gold, MD, director of dermatology clinical research at Henry Ford Health in Detroit, Michigan.

Stein Gold, principal investigator of the phase 3 LATITUDE Atlas trial, discussed head-to-head data presented at the 

2026 European Academy of Dermatology and Venereology (EADV) Congress

 in Vienna, Austria, in an interview with 

Why zasocitinib outperformed deucravacitinib in plaque psoriasis

Why Zasocitinib Outperformed Deucravacitinib in New Plaque Psoriasis Data

In LATITUDE Atlas, 36.5% of patients receiving zasocitinib achieved a 100% improvement in Psoriasis Area and Severity Index (PASI 100) at week 16, compared with 13.9% receiving deucravacitinib (

 < .001), according to Takeda.¹ Stein Gold noted the trial was the first head-to-head comparison of 2 drugs sharing the same mechanism of action, and the first to use PASI 100 as its primary endpoint. Based on the data so far, she said zasocitinib appears to perform similarly to traditional biologics.

What Explains the Efficacy Gap Between 2 TYK2 Inhibitors?

Despite their shared mechanism, Stein Gold described the 2 agents as quite different. She cited more than 1 million-fold greater selectivity for TYK2 over Janus kinase (JAK) 1, 2, and 3 with zasocitinib, compared with roughly 87-fold with deucravacitinib, a distinction aimed at avoiding off-target metabolic or hematologic effects.¹ She also pointed to the duration of interleukin (IL)-23 blockade, which extends over 24 hours with zasocitinib versus just under 5 hours with deucravacitinib.

Higher rates of complete clearance with zasocitinib emerged as early as week 8.² Stein Gold explained oral agents have historically taken time to show an effect, which has required considerable reassurance for patients. Visible improvement within the first several weeks gives patients confidence to continue treatment, even before their skin is fully clear.

In the pivotal LATITUDE PsO 3001 and 3002 studies, up to 93% of week 24 responders maintained skin clearance through week 52 or week 40, and patients reported improvements in itch and quality of life as early as week 2.¹ Stein Gold emphasized itch as a central component of psoriasis for many patients, contributing to poor sleep and diminished quality of life. The goal, in her view, is for patients to live as people with psoriasis rather than living their lives around the disease.

Patients want to gain control of their disease and keep it, Stein Gold added, and they need confidence their medication can deliver both over time.

Where Does Zasocitinib Fit in Psoriasis Treatment?

Stein Gold noted clinicians have not always presented an oral option to patients with moderate to severe plaque psoriasis, since no oral drug previously combined adequate efficacy, tolerability, and safety. With next-generation TYK2 inhibitors and other recently approved oral agents, she now offers these medications as a first-line option for patients not responding to topical therapy. Zasocitinib remains under review by the US Food and Drug Administration and the European Medicines Agency.¹

The most common adverse events across the LATITUDE program were upper respiratory tract infection, nasopharyngitis, and acne.¹ Stein Gold observed acne occurred more often with zasocitinib than with placebo and slightly more often than with deucravacitinib, although most cases were mild to moderate and many resolved without treatment. She recommended counseling patients up front about the possibility of acne, noting dermatologists can manage it, often with topical therapy alone.

Editor’s note: This summary has been edited for grammar and clarity using artificial intelligence tools. Financial disclosures reported for Stein Gold include AbbVie, Amgen, Arcutis, Bristol Myers Squibb, Organon Health, Eli Lilly, Johnson & Johnson, Novartis, Pfizer, and UCB.

Takeda. Zasocitinib delivered superior efficacy versus deucravacitinib in phase 3 LATITUDE psoriasis studies. News release. Published September 30, 2026. Accessed October 6, 2026. 

https://www.takeda.com/newsroom/news-releases/2026/zasocitinib-superior-efficacy/

HCPLive. EADV 2026: Zasocitinib tops deucravacitinib for PASI 100 in plaque psoriasis. Published September 30, 2026. Accessed October 6, 2026. 

https://www.hcplive.com/view/eadv-2026-zasocitinib-tops-deucravacitinib-for-pasi-100-in-plaque-psoriasis

Videos

Linda Stein Gold, MD, discusses LATITUDE Atlas data showing zasocitinib achieved higher PASI 100 rates than deucravacitinib in psoriasis.

Conference

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

Edward Chen, MD, is a professor of surgery at Thomas Jefferson University and the chief of cardiac surgery at Jefferson Abington Hospital.

 care has been on an upward trajectory in 2026, with recent guideline updates and trial readouts providing new perspectives on the myriad issues within this specialty.

, Edward Chen, MD, professor of surgery at Thomas Jefferson University and chief of cardiac surgery at Jefferson Abington Hospital, discussed his perspective on the growth of aortic surgery and his expertise in its application.

Updates and Evolution in Aortic Disease Care, With Edward Chen, MD

“Over the last 25 years since I started my training in cardiac surgery, taking care of the patient after the operation has really led to significant improvement in the outcomes of these types of operations,” Chen told 

. “Whereas before it might have been a coin flip, and it would be hard to know if someone’s going to do well, now, with the right patient selection and thorough evaluation of all of the variables, we can be very confident that many of these patients can go on to not only normal recovery, but a normal life.”

Chen’s statements on this patient-first approach mirror the recently published European Society for Vascular Surgery (ESVS) 2026 Clinical Practice Guidelines, published in February 2026, which emphasize the importance of the patient-centered treatment process. In the guidelines, the authors suggest that clinicians expand their focus to include patient perspectives while tailoring interventions to fit life expectancy and functional status. Additionally, the document pushes for further inclusion of multidisciplinary care, centralizing treatment in high-volume centers and prioritizing team-based decision making in regard to care.

Aortic disease care is also evolving through ongoing clinical trials, including the ARISE III trial at Stanford University. The program focuses on a less invasive alternative for certain high-risk patients with de novo Type A aortic dissections, evaluating the efficacy and safety of a new investigational stent graft device. The trial began in May of 2026 and is currently ongoing with plans to conclude in 2030. ARISE III currently aims to enroll 112 patients across the US.

Jefferson Health is also participating in an ongoing trial investigating the efficacy and safety of the Navitor Transcatheter Aortic Valve Implantation (TAVI) system, which is in development by Abbott. Roughly 1500 patients will be enrolled in the trial at ≤80 sites in the US and ≤15 internationally. The device will be investigated in both patients with symptomatic, severe native aortic stenosis at intermediate or low risk of surgical mortality and in patients with symptomatic heart disease due to failure of a surgical or transcatheter bioprosthetic aortic valve at high or greater surgical risk.

Chen also discussed his move to Jefferson Health and his upcoming projects within the organization, highlighting several potential targets for research.

“A lot of this work involves looking at aortic root surgery outcomes related to valve repair, aortic arch operations, various methods for protection – not just what I do, but also what my colleagues do in microval repair, coronary artery bypass, transplantation, and so on,” Chen said. “There’s a wide breadth of research going on here, and really, I’m only just scratching the surface of what’s being done.”

Editors’ Note: Chen reports no relevant disclosures.

Wanhainen A, Gombert A, Antoniou GA, et al. European Society for Vascular Surgery (ESVS) 2026 Clinical Practice Guidelines on the Management of Descending Thoracic and Thoraco-Abdominal Aortic Diseases - Editor's Choice. 

Nichols L. Stanford Continues to Advance Ascending Aorta Treatments Through National ARISE III Clinical Trial. 

Stanford Department of Cardiothoracic Surgery

. July 30, 2026. Accessed October 6, 2026. 

https://med.stanford.edu/ctsurgery/about-the-department/news/2026/stanford-continues-to-advance-ascending-aorta-treatments.html

Abbott Medical Devices. ENVISION IDE Trial: Safety and Effectiveness of NAVITOR in Transcatheter Aortic Valve Implantation (ENVISION). ClinicalTrials.gov Identifier: NCT05932615. Updated September 11, 2026. Accessed October 6, 2026. 

https://clinicaltrials.gov/study/NCT05932615#contactlocation

Chen discusses his experience with aortic and cardiac surgery, as well as ongoing research into ways to optimize and improve care.

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...

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Hisamitsu Hayashi, PhD, in the Graduate School of Pharmaceutical Sciences associate professors at the University of Tokyo.

For US clinicians managing progressive familial intrahepatic cholestasis (PFIC), approved drug therapy targets the itch, not the disease. The 

 has approved the ileal bile acid transporter (IBAT) inhibitor odevixibat (Bylvay) to treat pruritus in patients aged ≥3 months with PFIC.¹ Yet most patients still develop fibrosis and end-stage 

PFIC is a heterogeneous group of autosomal recessive disorders of childhood in which defects in hepatocellular transport genes disrupt bile formation.² Estimated incidence ranges from 1 in 50,000 to 1 in 100,000 births, and PFIC accounts for 10% to 15% of childhood cholestasis cases and 

10% to 15% of pediatric liver transplant indications

PFIC1, caused by ATP8B1 mutations, and PFIC2, caused by ABCB11 mutations affecting the bile salt export pump (BSEP), usually appear in the first months of life with jaundice and severe pruritus.² Serum gamma-glutamyltransferase (GGT) activity is normal in both, a key clue distinguishing them from PFIC3, and diagnosis is confirmed through genetic testing.² PFIC1 can carry extrahepatic features including short stature, sensorineural deafness, diarrhea, and pancreatitis, while patients with PFIC2, particularly those with biallelic truncating mutations, face an estimated 15% risk of hepatocellular carcinoma or cholangiocarcinoma.²

A different approach now has regulatory backing. In August 2026, sodium phenylbutyrate (Buphenyl), a longstanding urea cycle disorder therapy, gained approval in Japan for PFIC types 1 and 2 based on a series of clinical studies and an investigator-initiated trial.³ Rather than targeting pruritus, the drug increases functional BSEP at the canalicular membrane to restore bile acid export.

Hisamitsu Hayashi, PhD, associate professor in the Laboratory of Molecular Pharmacokinetics at the University of Tokyo Graduate School of Pharmaceutical Sciences, led the laboratory work identifying the drug's effect on BSEP. Following PFIC Awareness Day on October 5, Hayashi spoke with HCPLive in a written interview about early diagnosis, the evidence for a disease-directed therapy, and how it could complement IBAT inhibitors.

Q&A: Restoring bile acid export in PFIC types 1 and 2

HCPLive: What should clinicians know about PFIC types 1 and 2, and what unmet needs remain for patients?

 PFIC types 1 and 2 are rare inherited cholestatic liver diseases caused by defects in ATP8B1 and ABCB11, respectively. Both typically present in infancy or childhood with cholestasis and severe pruritus, but importantly, they are progressive liver diseases. Over time, patients may develop fibrosis, cirrhosis, and ultimately require liver transplantation.

Pruritus has an enormous impact on quality of life, but controlling pruritus does not necessarily mean that progression of the underlying liver disease has been controlled. Therefore, we need to consider both symptom control and preservation of the native liver.

Now that medical treatment options are emerging, I think early diagnosis is becoming increasingly important. Rather than waiting until patients have advanced fibrosis or hepatic decompensation, we should aim to establish the molecular diagnosis and intervene as early as possible, when there may still be an opportunity to preserve liver function.

This also makes genetic diagnosis increasingly relevant, because PFIC is biologically heterogeneous and treatment response may depend on the underlying disease subtype and molecular defect.

HCPLive: How does sodium phenylbutyrate work in PFIC, and are some patients, such as those with particular genetic variants, more likely to respond?

 BSEP is located at the canalicular membrane of hepatocytes and exports bile acids from the liver into bile. Sodium phenylbutyrate increases the amount of functional BSEP at this membrane, in part by stabilizing BSEP that has reached the cell surface.

In PFIC2, some ABCB11 variants produce BSEP proteins that retain transport activity but have reduced expression at the canalicular membrane. For those patients, increasing the amount of functional BSEP at the membrane provides a clear mechanistic rationale for treatment.

However, sodium phenylbutyrate is not simply a mutation-specific chaperone. We originally found that it also increases the membrane expression and transport capacity of wild-type BSEP. This is particularly relevant to PFIC1, where ABCB11 itself is not mutated, but ATP8B1 deficiency can lead to secondary impairment of BSEP function. Enhancing functional wild-type BSEP may therefore improve bile acid excretion even when BSEP is not the primary genetic defect.

Biologically, this suggests that treatment response may depend on how much recoverable BSEP function remains. However, we do not yet have a validated genotype-based predictor of response, and the Japanese approval is not restricted to particular variants.

The effect on wild-type BSEP also raises the possibility that this mechanism could be relevant to other cholestatic conditions in which canalicular BSEP function is secondarily reduced. That is scientifically interesting, but at present this remains a research question; the approved indication is PFIC types 1 and 2.

HCPLive: How do you see sodium phenylbutyrate fitting alongside current options such as IBAT inhibitors, biliary diversion, and liver transplant?

 I think an important point is that the approved indications and therapeutic roles are different.

In Japan, IBAT inhibitors are approved specifically for the treatment of cholestatic pruritus associated with PFIC, not for PFIC itself as the approved indication. The approved indications are also pruritus-focused in the US, although regulatory labeling differs across regions.

Sodium phenylbutyrate, by contrast, is approved for PFIC types 1 and 2 themselves. It acts in the liver by increasing functional BSEP expression at the canalicular membrane and enhancing bile acid export from hepatocytes. From the current regulatory and clinical perspective, I therefore view sodium phenylbutyrate as a therapy directed at the underlying disease mechanism, whereas IBAT inhibitors are positioned primarily as treatments for cholestatic pruritus.

That distinction does not mean that IBAT inhibitors have no effect on disease biology. By reducing intestinal bile acid reabsorption and the circulating bile acid pool, they may have broader biological effects. But their approved indication in PFIC is cholestatic pruritus, whereas sodium phenylbutyrate is approved for PFIC1 and PFIC2 themselves.

Because they act at different points in bile acid physiology, these treatments may ultimately be complementary rather than competing. Future studies should determine how best to select, sequence, or potentially combine them according to disease subtype, genotype, residual BSEP function, and disease stage.

Biliary diversion remains an important option when medical therapy is insufficient, and liver transplantation remains essential for patients with advanced or decompensated liver disease. This is also why early diagnosis and early intervention are becoming increasingly important: once irreversible liver damage has developed, the opportunity for medical therapy to preserve the native liver becomes much more limited.

HCPLive: What did the clinical evidence supporting the approval show, and which outcomes do you think are most meaningful for patients?

 The multicenter investigator-initiated study enrolled 6 patients with PFIC2. The primary endpoint was change in liver histology at 24 weeks. Histological improvement was observed in 3 of the 6 patients, and liver biochemical parameters generally improved during treatment. PMDA [Pharmaceuticals and Medical Devices Agency] considered these findings meaningful in the context of a progressive disease in which spontaneous histological improvement would not generally be expected.

What I find particularly important is the long-term follow-up. Four patients who continued treatment were still receiving sodium phenylbutyrate as of March 2026 and had survived with their native livers for approximately 6.5 to 8 years. In our nationwide survey of Japanese patients with PFIC2, the historical disease course was characterized by frequent progression to liver transplantation at a young age. Against that background, the duration of native liver survival observed in the treated patients appears substantially longer than the clinical course we had historically observed in Japanese patients with PFIC2. Of course, this is not a controlled comparison, and the number of patients is very small, so we have to be cautious.

For PFIC1, the evidence came from an exploratory study in 3 patients. Pruritus improved during treatment and worsened again after discontinuation, providing supportive evidence of clinical benefit, although histological changes were not clearly demonstrated during the short treatment period.

For patients and families, I think the most meaningful outcomes are not laboratory values alone. They are relief from severe pruritus, preservation of growth and quality of life, prevention of progressive liver injury, and ultimately the ability to remain well with one's native liver for as long as possible.

HCPLive: Can you walk us through the path from your lab's initial sodium phenylbutyrate findings to its approval?

 Our starting point was not drug repurposing itself, but understanding the disease mechanism.

We were studying how impaired BSEP expression and function at the canalicular membrane contribute to cholestasis in PFIC. Our pathophysiological studies showed that some PFIC2-associated BSEP variants retain transport activity but have impaired trafficking to, or reduced stability at, the canalicular membrane. This led us to ask whether restoring functional BSEP expression at the hepatocyte membrane could become a therapeutic strategy.

In the course of that work, we discovered that sodium phenylbutyrate has a previously unrecognized pharmacological action that is completely different from its established nitrogen-scavenging effect in urea cycle disorders: it increases functional BSEP expression at the canalicular membrane and enhances bile acid transport.

We then moved step by step from mechanistic studies to translational research. We demonstrated the effect in experimental models and human liver samples, conducted exploratory clinical studies in both PFIC1 and PFIC2, and subsequently performed a multicenter investigator-initiated clinical trial in patients with PFIC2.

The development therefore followed a path from understanding disease biology, to discovering a new pharmacological action, to testing that mechanism in patients. Approximately 20 years after the initial discovery, sodium phenylbutyrate was approved in Japan in August 2026 for both PFIC types 1 and 2.

This long path required sustained collaboration among patients and families, clinicians, researchers, public funding agencies, and industry, and ultimately led to the regulatory submission by OrphanPacific.

HCPLive: What should US clinicians take away from this approval, and what research is needed next?

 I hope there are 2 major take-home messages.

The first is the importance of basic research into disease mechanisms. This development began with a very fundamental question: why does bile acid transport fail in PFIC? By studying the molecular and cellular mechanisms of the disease, we discovered an unexpected pharmacological action of an existing drug. That basic discovery eventually became a treatment for patients more than 2 decades later.

To me, this is an important example of how deep mechanistic research can reveal therapeutic opportunities that would not necessarily emerge from conventional drug screening or from the known pharmacology of an existing drug.

The second lesson is that successful development in rare diseases requires a sustainable ecosystem that extends beyond a single drug.

Based on what we learned during the development of sodium phenylbutyrate, we established CIRCLe, a research and clinical-development platform for rare disorders and undiagnosed conditions presenting with liver abnormalities in Japan. CIRCLe has already supported the development and post-marketing evaluation of IBAT inhibitors in Japan.

We are now also collaborating with other pharmaceutical companies on drug-development projects involving a broader range of rare and undiagnosed disorders with diverse liver manifestations, extending well beyond PFIC and cholestatic disease. CIRCLe is also expanding internationally through collaborations with medical institutions outside Japan.

We hope CIRCLe can serve not only as a platform for one disease or one therapeutic area, but as an infrastructure that helps accelerate the development of new treatments for rare disorders and undiagnosed conditions involving the liver more broadly.

For sodium phenylbutyrate itself, the next priorities include international validation, better understanding of genotype-response relationships, long-term evaluation of native liver survival, and determining how this therapy should be used alongside IBAT inhibitors and other emerging treatments.

Ultimately, our goal is not simply to develop one successful drug. It is to create an environment in which the knowledge gained from one successful development can make the next treatment easier to bring to patients.

Editor's Note: This transcript has been edited for grammar and clarity using artificial intelligence tools. Disclosures for Hayashi were not available at the time of publication.

Walter K. FDA approves odevixibat for PFIC. HCPLive. Published July 20, 2021. Accessed October 6, 2026. 

https://www.hcplive.com/view/fda-approves-odevixibat-pfic

Davit-Spraul A, Gonzales E, Baussan C, et al. Progressive familial intrahepatic cholestasis. 

Approved US PFIC drugs treat pruritus. The researcher behind a therapy targeting BSEP on diagnosis, data, and what comes next.

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