HCPLive Editorial Staff

Welcome to The HCPFive, your go-to roundup for the latest healthcare news and breakthroughs, curated specifically for busy healthcare professionals.

Each week, we highlight 5 key developments or headlines from healthcare that you need to know—whether it's a cutting-edge treatment, regulatory updates, or innovations shaping the future of medicine. This week's top stories include the US Food and Drug Administration (FDA) removal of an advisory committee requirement for the supplemental New Drug Application (sNDA) for sparsentan (Filspari) in 

focal segmental glomerulosclerosis (FSGS)

, Intercept Pharmaceuticals’ voluntary withdrawal of obeticholic acid (Ocaliva) from the US market, the FDA’s acceptance of a Letter of Intent for the qualification of FibroScan as a reasonably likely surrogate endpoint in clinical trials for 

metabolic dysfunction-associated steatohepatitis (MASH)

, the Agency’s clearance of a clinical trial evaluating a genetically engineered porcine-derived kidney in patients with 

, and positive data for Regeneron’s first-in-class investigational 

With The HCPFive, you'll get the essential takeaways to stay informed and ahead of the curve. Here’s your quick dive into the top stories for the week of September 7, 2025—let’s jump in!

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

On September 10, 2025, Travere Therapeutics announced that the FDA informed the Company that following further review of the sNDA for sparsentan (Filspari) in FSGS, an advisory committee is no longer needed. The sNDA remains under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of January 13, 2026. If approved, sparsentan would be the first medication indicated for FSGS.

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

On September 11, 2025, Intercept Pharmaceuticals announced its decision to voluntarily withdraw obeticholic acid (Ocaliva) from the US market for the treatment of PBC following a request from the FDA.The Agency has placed a clinical hold on all Intercept clinical trials conducted under a US IND involving obeticholic acid.

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

On September 8, 2025, the FDA’s Center for Drug Evaluation and Research, Office of New Drugs accepted Echosense’ Letter of Intent for the qualification of Liver Stiffness Measurement (LSM) by Vibration-Controlled Transient Elastography (VCTE; FibroScan) as a reasonably likely surrogate endpoint in clinical trials for MASH. The Letter of Intent included official letters of support from Eli Lilly, Boehringer Ingelheim, and Novo Nordisk.

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

On September 8, 2025, eGenesis announced the FDA clearance of its IND application to initiate a clinical trial evaluating EGEN-2784, a genetically engineered porcine-derived kidney, in patients with ESKD. The IND supports a phase 1/2/3 study designed to assess the safety, tolerability, and efficacy of EGEN-2784 at 24 weeks post-transplant in patients with ESKD who are ≥ 50 years of age, dialysis-dependent, and on the kidney transplant waitlist.

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

On September 8, 2025, Regeneron Pharmaceuticals announced results evaluating its first-in-class investigational allergen-blocking antibodies in allergen-challenge phase 3 trials in adults with moderate-to-severe cat or birch allergies. Both trials met their respective primary and key secondary endpoints. Data from the phase 3 trials will be presented at an upcoming medical conference and will inform confirmatory phase 3 trials.

Articles

Travere Therapeutics' sparsentan (Filspari) for FSGS is under FDA review without an advisory committee, with a PDUFA date set for January 2026. Intercept Pharmaceuticals has withdrawn obeticholic acid (Ocaliva) for PBC from the US market following FDA's request. The FDA accepted a Letter of Intent for FibroScan as a surrogate endpoint in MASH trials. eGenesis received FDA clearance for a clinical trial of a genetically engineered pig kidney for ESKD. Regeneron's allergen-blocking antibodies showed positive results in phase 3 trials for cat and birch allergies.

Stay updated with the latest healthcare breakthroughs, including FDA news and the voluntary withdrawal of obeticholic acid, in this week's essential news roundup.

The HCPFive: Top News for Healthcare Providers from the Week of 09/07

Among dermatologists and other clinicians in the skin health space, the 

European Academy of Dermatology and Venereology (EADV) Congress

 is more than just a yearly meeting—it is a venue at which new data can shift existing standards of care and redefine treatment approaches. The late-breaking abstract sessions each year during the EADV Congress bring forward findings that can help to not only inform decision-making but also spark debate, collaboration, and momentum across 

The 2025 EADV Congress, set to take place in Paris, France from September 17–20, is set to release a series of late-breaking findings spanning inflammatory skin disease and pigmentary disorders, with data that could impact both frontline practice and long-term research trajectories. These sessions will be an opportunity to gain firsthand insight into the science that will guide tomorrow’s treatment landscape for conditions such as 

, atopic dermatitis, alopecia areata, and more.

 team has identified 13 phase 2 and phase 3 late-breaking presentations for clinicians to watch at EADV 2025. These studies highlight the evolving innovation pipeline across dermatology and venereology. 

1. Long-term efficacy and complete scalp hair regrowth in patients with alopecia areata receiving ritlecitinib 50 mg QD up to 3 years in the ALLEGRO clinical trial program

Presentation Time: Thursday, September 18, 14:15 - 14:30 CEST
Presenter: Brett King, MD, PhD
Background Info: 

 New long-term findings from the ALLEGRO program will highlight durability of response with ritlecitinib (Litfulo) in alopecia areata, with data extending out to 3 years. The analysis focuses on sustained scalp hair regrowth and the proportion of patients achieving complete response over time with the agent, which received approval from the FDA for treatment of severe alopecia areata in adults and adolescents 12 years and older in June 2023.

2. Povorcitinib for Moderate to Severe Hidradenitis Suppurativa: Week 24 Interim Phase 3 Results

Presentation Time: Wednesday, September 17, 14:45 - 15:00 CEST
Presenter: Martina Porter, MD
Background Info: 

This phase 3 interim analysis will present 24-week efficacy and safety data for povorcitinib in patients with moderate to severe hidradenitis suppurativa. Results will provide insight into the potential role of this investigational JAK1 inhibitor in addressing an area of high unmet clinical need.

In March 2025, Incyte announced both of the phase 3 STOP-HS trials, STOP-HS1 and STOP-HS2, met their primary endpoint at both tested doses (45 mg and 75 mg), with a significantly higher proportion of patients treated with povorcitinib once daily versus placebo achieved Hidradenitis Suppurativa Clinical Response.

3. Safety of delgocitinib cream in adult patients with Chronic Hand Eczema (CHE): pooled analysis of five phase 2b and phase 3 trials

Presentation Time: Thursday, September 18, 15:15 - 15:30 CEST
Presenter: Robert Bissonnette, MD
Background Info: 

A pooled analysis of 5 phase 2b and phase 3 trials will evaluate the safety of delgocitinib cream (Anzupgo) in adults with chronic hand eczema (CHE). The agent, an inhibitor of JAK1 and TYK2, received a historic approval in July 2025 as the first FDA-approved therapy for CHE. 

The results of the EADV analysis aim to clarify its tolerability and support its potential as a long-term topical treatment option for this challenging condition.

4. Baricitinib in the Treatment of Adults with Pyoderma Gangrenosum: A Phase II Open Label Trial

Presentation Time: Wednesday, September 17, 14:30 - 14:45 CEST
Presenter: Alex Ortega Loayza, MD
Background Info: 

This phase 2 open-label trial will assess the efficacy and safety of baricitinib (Olumiant) in adults with pyoderma gangrenosum. The study aims to explore the agent, which boasts approvals for rheumatoid arthritis and alopecia dating back to 2018 and 2022, respectively, in managing this rare and difficult-to-treat inflammatory skin disorder.

5. Efficacy and safety of izokibep, a novel interleukin-17A inhibitor, in moderate to severe hidradenitis suppurativa: Week 16 results from a randomised, double-blind, placebo-controlled, multicentre, phase 3 study

Presentation Time: Wednesday, September 17, 15:00 - 15:15 CEST
Presenter: Kim A. Papp, MD, PhD
Background Info:

 Week 16 results from this phase 3 trial will detail the efficacy and safety of izokibep, a novel small interleukin-17A inhibitor, in patients with moderate to severe hidradenitis suppurativa. In August 2024, Acelyrin announced izokibep demonstrated statistically signiﬁcant responses across multiple eﬃcacy endpoints at 12 weeks, including the trial's primary outcome of HiSCR75.

Data from the study will add to the growing body of evidence on IL-17 blockade as a therapeutic strategy in this challenging disease.

6. IL-22RA1 antagonism with temtokibart provides significant early and sustained improvements in atopic dermatitis: results from a phase 2b dose-finding trial

Presentation Time: Wednesday, September 17, 15:15 - 15:30 CEST
Presenter: Stephan Weidinger, MD, PhD
Background Info: 

This phase 2b dose-finding trial will present data on temtokibart, an IL-22RA1 antagonist, showing early and sustained improvements in patients with atopic dermatitis. In May 2025, LEO Pharma announced positive topline data from the trial indicating use of temtokibart was associated with improvement in achieved the primary endpoint of percent change in Eczema Area and Severity Index (EASI) score. The results aim to highlight the potential of targeting IL-22 signaling as a novel approach in managing moderate-to-severe disease.

7. Temtokibart, an IL-22RA1 Monoclonal Antibody broadly dampens gene expression markers of activated immune pathways in Atopic Dermatitis: Results from a Phase 2b Trial Subgroup Analysis

Presentation Time: Wednesday, September 17, 15:30 - 15:45 CEST
Presenter: Ester Del Duca, MD
Background Info: 

This subgroup analysis will explore how data from the phase 2b dose-finding trial demonstrates temtokibart's modulation of gene expression and its association with activated immune pathways in atopic dermatitis. The findings aim to shed light on the molecular mechanisms underlying its clinical efficacy, which will be integral to its integration in treatment algorithms for the heterogenous disease.

8. Efficacy and Safety of Ruxolitinib Cream in Patients With Prurigo Nodularis: Pooled Results From the Phase 3 TRuE-PN1 and TRuE-PN2 Randomized, Vehicle-Controlled Studies

Presentation Time: Wednesday, September 17, 16:00 - 16:15 CEST
Presenter: Sonja Ständer, MD
Background Info: 

Pooled phase 3 data will assess the efficacy and safety of ruxolitinib cream 0.15% (Opzelura) in patients with prurigo nodularis. The TRuE-PN1 and TRuE-PN2 trials captured the attention of the dermatology community in March 2025 when Incyte announced the TRuE-PN1 trial had met its primary endpoint, but the TRuE-PN2 trial failed to achieve statistical significance for the same outcome due to high placebo response. The results are expected to provide a comprehensive view of its potential as a targeted topical therapy for this chronic, intensely pruritic condition.

9. Maintenance of Response With Icotrokinra, a Targeted Oral Peptide, for the Treatment of Moderate-to-Severe Plaque Psoriasis: Randomized Treatment Withdrawal in Adults (Weeks 24-52) and Continuous Treatment in Adolescents (Through Week 52) From the Phase 3, ICONIC-LEAD Trial

Presentation Time: Wednesday, September 17, 16:15 - 16:30 CEST
Presenter: Jennifer Soung, MD
Background Info:

 The ICONIC-LEAD trial will present data on the durability of response with icotrokinra in adults and adolescents with moderate-to-severe plaque psoriasis. ICONIC-LEAD is a multicenter, randomized, double-blind, placebo-controlled study, with 684 participants aged 12 years and older enrolled at 170 sites globally. Icotrokinra is a first-in-class, targeted oral peptide designed to selectively bind the interleukin (IL)-23 receptor and inhibit IL-23 pathway signaling. These findings will highlight both treatment withdrawal and continuous therapy to explore long-term disease control.

10. Bimekizumab efficacy and safety through 3 years in patients with hidradenitis suppurativa: Results from the phase 3 BE HEARD I&II trials and their open-label extension BE HEARD EXT

Presentation Time: Wednesday, September, 17:00 - 17:15 CEST
Presenter: John R. Ingram, MD, PhD
Background Info: 

Long-term data from BE HEARD I&II and the open-label extension will assess the sustained efficacy and safety of bimekizumab in hidradenitis suppurativa over three years. Bimekizumab is the first and only approved medication designed to selectively inhibit both IL-17A and IL-17F among patients. These new results may help to inform long-term management strategies for this chronic condition.

11. Molecular and Clinical Effects of Oral ATI-2138, an ITK/JAK3 inhibitor, in Moderate-to-Severe Atopic Dermatitis: Sub-study of a Phase 2a Open-Label, Single-Arm Trial

Presentation Time: Thursday, September 18, 14:15 - 14:30 CEST
Presenter: Jessica Beaziz Tordjman, MD
Background Info: 

This sub-study evaluates both molecular and clinical outcomes of oral ATI-2138 in moderate-to-severe atopic dermatitis. ATI-2138 is a novel pharmacologic agent which acts as a dual inhibitor of interleukin-2-inducible T cell kinase (ITK) and JAK3. These late-breaking findings may provide insights into how ITK/JAK3 inhibition translates to disease improvement at the cellular level.

12. DELTA TEEN Phase 3 trial: Efficacy and Safety of Delgocitinib Cream in Adolescents with Moderate to Severe Chronic Hand Eczema

Presentation Time: Thursday, September 18, 14:30 - 14:45 CEST
Presenter: Sonja Molin, MD
Background Info: 

The DELTA TEEN trial will report on the efficacy and safety of delgocitinib cream in adolescents with chronic hand eczema. DELTA TEEN is a 16-week, phase 3 DELTA TEEN trial that used a randomized, double-blind, vehicle-controlled, parallel-group, multi-site design. Investigators explored delgocitinib's efficacy and safety with twice-daily use. Their primary endpoint was an Investigator’s Global Assessment for chronic hand eczema treatment success (IGA-CHE TS) at Week 16. These data may help to expand treatment options for this age group in a condition that is often challenging to manage.

13. Initial “Super Response” to Tralokinumab Leads to Stable Long-term Response in Patients with Moderate-to-Severe Atopic Dermatitis: Responder and Predictor Analysis from the ECZTRA 3 & ECZTEND Trials

Presentation Time: Friday, September 19, 15:30 - 15:45 CEST
Presenter: Andrew Blauvelt, MD
Background Info: 

This analysis will explore how early “super responders” to tralokinumab maintain long-term control of moderate-to-severe atopic dermatitis. The 32-week ECZTRA 3 study assessed the effectiveness and safety of tralokinumab plus topical corticosteroids (TCS) versus placebo plus TCS. ECZTEND was an open-label, single-arm, multi-center, long-term extension study designed to assess the tralokinumab in patients with atopic dermatitis who had participated in prior tralokinumab clinical research. These late-breaking data will examine predictors of sustained response, potentially guiding patient selection and treatment optimization.

For more information from the EADV Congress 2025 and other conference coverage, view our 

Editor's note: The summaries of these data were edited with the help of AI tools.

Long-term efficacy and complete scalp hair regrowth in patients with alopecia areata receiving ritlecitinib 50 mg QD up to 3 years in the ALLEGRO clinical trial program. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Povorcitinib for Moderate to Severe Hidradenitis Suppurativa: Week 24 Interim Phase 3 Results. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Safety of delgocitinib cream in adult patients with Chronic Hand Eczema (CHE): pooled analysis of five phase 2b and phase 3 trials. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Baricitinib in the Treatment of Adults with Pyoderma Gangrenosum: A Phase II Open Label Trial. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Efficacy and safety of izokibep, a novel interleukin-17A inhibitor, in moderate to severe hidradenitis suppurativa: Week 16 results from a randomised, double-blind, placebo-controlled, multicentre, phase 3 study. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

IL-22RA1 antagonism with temtokibart provides significant early and sustained improvements in atopic dermatitis: results from a phase 2b dose-finding trial. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Temtokibart, an IL-22RA1 Monoclonal Antibody broadly dampens gene expression markers of activated immune pathways in Atopic Dermatitis: Results from a Phase 2b Trial Subgroup Analysis. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Efficacy and Safety of Ruxolitinib Cream in Patients With Prurigo Nodularis: Pooled Results From the Phase 3 TRuE-PN1 and TRuE-PN2 Randomized, Vehicle-Controlled Studies. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Maintenance of Response With Icotrokinra, a Targeted Oral Peptide, for the Treatment of Moderate-to-Severe Plaque Psoriasis: Randomized Treatment Withdrawal in Adults (Weeks 24-52) and Continuous Treatment in Adolescents (Through Week 52) From the Phase 3, ICONIC-LEAD Trial. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Bimekizumab efficacy and safety through 3 years in patients with hidradenitis suppurativa: Results from the phase 3 BE HEARD I&II trials and their open-label extension BE HEARD EXT. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Molecular and Clinical Effects of Oral ATI-2138, an ITK/JAK3 inhibitor, in Moderate-to-Severe Atopic Dermatitis: Sub-study of a Phase 2a Open-Label, Single-Arm Trial. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

DELTA TEEN Phase 3 trial: Efficacy and Safety of Delgocitinib Cream in Adolescents with Moderate to Severe Chronic Hand Eczema. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

Initial “Super Response” to Tralokinumab Leads to Stable Long-term Response in Patients with Moderate-to-Severe Atopic Dermatitis: Responder and Predictor Analysis from the ECZTRA 3 & ECZTEND Trials. Presented at: EADV Congress 2025. September 17 – 20, 2025. Paris, France.

The European Academy of Dermatology and Venereology (EADV) Congress is a pivotal event for dermatologists, offering new data that can redefine treatment standards. The 2025 Congress in Paris will feature late-breaking findings on inflammatory skin diseases and pigmentary disorders, potentially impacting clinical practice and research. Key studies include trials on ritlecitinib for alopecia areata, povorcitinib for hidradenitis suppurativa, and delgocitinib cream for chronic hand eczema. These studies highlight innovations in dermatology, focusing on long-term efficacy, safety, and novel therapeutic strategies for challenging skin conditions.

A look at 13 phase 2 and phase 3 studies with significant implications from EADV 2025's late-breaking sessions.

EADV Congress 2025: Late-Breaking Trials Preview

Each week, we highlight 5 key developments or headlines from healthcare that you need to know—whether it's a cutting-edge treatment, regulatory updates, or innovations shaping the future of medicine. This week's top stories include the US Food and Drug Administration (FDA) extension of mitapivat’s Prescription Drug User Fee Act (PDUFA) date for its supplemental New Drug Application (sNDA) in 

, an sNDA submission for roflumilast cream 0.3% (Zoryve) for 

 in pediatric patients as young as 2 years of age, zilebesiran’s progression to a phase 3 clinical trial for 

, positive phase 1b/2a RestorAATion-2 data evaluating WVE-006 as a treatment for 

, and findings from the phase 3 COAST 1 trial of amlitelimab in 

With The HCPFive, you'll get the essential takeaways to stay informed and ahead of the curve. Here’s your quick dive into the top stories for the week of August 31, 2025—let’s jump in!

Mitapivat PDUFA for Thalassemia Delayed to December

On September 4, 2025, the FDA extended the PDUFA date for Agios’ supplemental New Drug Application (sNDA) of mitapivat for the treatment of adult patients with non-transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia by 3 months to December 7, 2025.

The oral pyruvate kinase (PK) activator is currently approved for treating adults with PK deficiency under the name Pyrukynd. Data supporting the sNDA submission for its use in NTD and TD alpha- or beta-thalassemia are from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T phase 3 trials.

Arcutis Submits sNDA for Roflumilast Cream 0.3% for Psoriasis in Children Aged 2-5 Years

On September 3, 2025, Arcutis Biotherapeutics submitted an sNDA to the FDA for roflumilast cream 0.3% (Zoryve) for plaque psoriasis in pediatric patients as young as 2 years of age, potentially expanding the treatment’s indication. If roflumilast cream’s sNDA clears and the age limit is expanded, it would be the first and only topical phosphodiesterase 4 (PDE4) inhibitor indicated for psoriasis among patients in this age group.

Zilebesiran, RNAi for Hypertension, Heads to Phase 3 Cardiovascular Outcomes Trial

Alnylam Pharmaceuticals announced plans to advance zilebesiran, their subcutaneously administered RNA-interfering therapeutic, into a phase 3 cardiovascular outcomes trial in patients with uncontrolled hypertension. Coined as ZENITH, the trial design is based on data from the KARDIA phase 2 program, including the KARDIA-3 trial, which missed its primary endpoint but met the objective of informing the design, patient population, and dose and was presented at the 

European Society of Cardiology (ESC) Congress 2025

RestorAATion-2: WVE-006 Achieves Durable Serum AAT Protein Production in AATD

On September 3, 2025, Wave Life Sciences announced positive data from the 200 mg single and multidose and 400 mg single dose cohorts of the ongoing phase 1b/2a RestorAATion-2 study evaluating WVE-006 as a treatment for AATD. In the trial, WVE-006 achieved durable production of serum AAT protein at levels associated with a reduced risk of AATD liver and lung diseases following repeat 200 mg doses. While data from the 400 mg multidose cohort are not expected until 1Q 2026, single dose 400 mg data showed achievement of 12.8 µM total AAT and 5.3 µM M-AAT.

Phase 3 Data Show Amlitelimab Effective in Adults, Adolescents with Atopic Dermatitis

On September 4, 2025, Sanofi announced positive data from the global COAST 1 trial evaluating amlitelimab, a fully human, non–T-cell depleting monoclonal antibody directed against OX40-ligand, in patients ≥ 12 years of age with moderate-to-severe atopic dermatitis. In the trial, amlitelimab treatment administered every 4 or every 12 weeks led to significant skin clearance and reduction in severity versus placebo at week 24.

The FDA has extended the PDUFA date for mitapivat's sNDA in thalassemia to December 2025. Arcutis Biotherapeutics has submitted an sNDA for roflumilast cream for pediatric psoriasis, potentially expanding its indication. Alnylam Pharmaceuticals is advancing zilebesiran to a phase 3 trial for uncontrolled hypertension. Wave Life Sciences reported positive data from the RestorAATion-2 study on WVE-006 for AATD. Sanofi announced positive results from the COAST 1 trial of amlitelimab for atopic dermatitis, showing significant efficacy in skin clearance and severity reduction.

Stay updated with the latest healthcare breakthroughs, including FDA news and phase 3 clinical trial data, in this week's essential news roundup.

The HCPFive: Top News for Healthcare Providers from the Week of 08/31

Each week, we highlight 5 key developments or headlines from healthcare that you need to know—whether it's a cutting-edge treatment, regulatory updates, or innovations shaping the future of medicine. This week's top stories include the US Food and Drug Administration (FDA) Priority Review of omidubicel for 

, the approval of an updated Risk Evaluation and Mitigation Strategy (REMS) label for sparsentan (Filspari) for 

, a Complete Response Letter (CRL) issued to ONS-5010 for the treatment of wet 

, new American College of Cardiology guidance on respiratory disease vaccines for adults with 

, and phase 3 ATTAIN-2 trial data for orforglipron in adults with obesity or 

With The HCPFive, you'll get the essential takeaways to stay informed and ahead of the curve. Here’s your quick dive into the top stories for the week of August 24, 2025—let’s jump in!

FDA Accepts Priority Review Application of Omidubicel for Severe Aplastic Anemia

On August 25, 2025, the FDA accepted the priority review application for Ayrmid’s omidubicel, an investigative cell therapy, for the treatment of severe aplastic anemia. With the acceptance, the FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 10, 2025.

FDA Approves REMS Label Update for Sparsentan (Filspari) in IgA Nephropathy

On August 27, 2025, Travere Therapeutics announced the FDA approval of an updated REMS labeling for sparsentan (Filspari) for the treatment of IgAN, reducing the frequency of liver function monitoring from monthly to every 3 months from the onset of treatment with sparsentan and removing the embryo-fetal toxicity monitoring requirement.

FDA Issues CRL to Outlook Therapeutics' ONS-5010 for the Treatment of Wet AMD

On August 28, 2025, the FDA issued a CRL to Outlook Therapeutics' Biologics License Application for ONS-5010 for the treatment of wet AMD, citing the agent’s failure to achieve the primary efficacy endpoint in the phase 3 NORSE EIGHT trial. With the CRL, the FDA suggested the Company submit confirmatory evidence of efficacy to support the next application. The Agency did, however, reiterate the achievement of NORSE TWO's primary effectiveness endpoint.

ACC Releases Respiratory Disease Vaccination Guidelines for Adults with Heart Disease

On August 26, 2025, the American College of Cardiology released Concise Clinical Guidance recommending vaccines to protect adults with cardiovascular disease against a variety of respiratory diseases, including 

, respiratory syncytial virus (RSV), and 

. The guidelines also aim to improve access to vaccines, effectively circumventing financial barriers to access, providing accurate and practical education, and assuaging concerns that give rise to vaccine hesitancy and resistance.

Orforglipron Delivers Weight Loss, A1C Reductions in Phase 3 ATTAIN-2 Trial

On August 26, 2025, Eli Lilly and Company announced positive topline results from the phase 3 ATTAIN-2 trial evaluating orforglipron, an investigational oral GLP-1 receptor agonist, in adults with obesity or overweight and type 2 diabetes. In the trial, all 3 doses of orforglipron met the primary and all key secondary endpoints, delivering significant weight loss, meaningful A1C reductions, and improvements in cardiometabolic risk factors at 72 weeks. With the completion of ATTAIN-2, Lilly now has the full clinical data package required to initiate global regulatory submissions for orforglipron.

The FDA has accepted the priority review application for omidubicel, a cell therapy for severe aplastic anemia, with a PDUFA target date set. Sparsentan's REMS label update for IgA nephropathy now requires less frequent liver monitoring and removes embryo-fetal toxicity monitoring. A CRL was issued for ONS-5010 for wet AMD due to unmet efficacy endpoints. The ACC released guidelines for respiratory disease vaccinations in adults with cardiovascular disease. Orforglipron showed promising results in a phase 3 trial for weight loss and A1C reduction in type 2 diabetes patients.

Stay updated with the latest healthcare breakthroughs, including FDA news, vaccine guidelines, and orforglipron data, in this week's essential news roundup.