Jain is the medical director of the pediatric and young adult program for hemostasis/thrombosis and sickle cell disease at Loma Linda University Children’s Health.

Prophylaxis With von Willebrand Factor VIII Reduces Bleeding in Children, With Akshat Jain, MD, MPH

Prophylaxis with a plasma-derived von Willebrand factor VIII (pdVWF/FVIII) concentrate efficaciously reduces bleeding in patients <6 years with 

67th American Society of Hematology (ASH) Annual Meeting and Exposition

 by Akshat Jain, MD, MPH, the medical director of the pediatric and young adult program for hemostasis/thrombosis and sickle cell disease at Loma Linda University Children’s Health, these data fill a gap in VWD care. A prior study, WIL-31, demonstrated the efficacy and safety of pdVWF/FVIII in a 1:1 activity ratio in adult, adolescent, and child patients aged ≥6 years. Prior to WIL-33, however, very little data was available for prophylaxis with VWF in patients <6 years with severe VWD.

“Whenever a clinician is faced with a person with such a severe bleeding disorder, the challenge of guidance on how to treat that patient, how to monitor them, is always there,” Jain told 

 in an exclusive interview. “Through this truly global trial, with sites across Asia, Africa, Europe, and North and South America, we’ve truly created a global database.”

WIL-33 was an open-label, non-controlled, international, multicenter, prospective phase 3 study investigating the efficacy, immunogenicity, and safety of pdVWF/FVIII prophylaxis and pharmacokinetics in patients <6 years with severe VWD, defined as VWF ristocetin cofactor activity (VWF;RCo) <20%. pdVWF/FVIII was given at a dose of 30-50 international units (IU) per kg over 12 months, 2-3 times per week. The primary endpoint was total annualized bleeding rate (TABR) during prophylaxis, while safety and tolerability were assessed throughout the study.

A total of 12 patients were ultimately enrolled in the trial. Baseline median age was 2 years (range, 1-5), 4 patients (33.3%) had VWD type 2, and 8 patients (66.7%) had VWD type 3. The per-protocol robustness (PPR) set, a subset of the full analysis set (FAS), excluded 3 patients due to deviations from recommended dosing. The mean TABR in the FAS and PR set was 4.6 (standard deviation [SD] 6.1) and 2.7 (1.8), respectively. Mean TABR was 2.1 (1) and 5.8 (7.2) in patients with VWD type 2 and VWD type 3, respectively.

Of 56 total bleeds, >1/3 (22/56; 39%) during prophylaxis in the FAS set were attributed to allergic rhinitis in 1 patient. Mean spontaneous ABR was 0.9 (1.2) in the FAS and 1 (1.3) in the PPR set. The median weekly prophylactic dose was 100 (range, 63-311) and 92 (63-130) IU/kg in the FAS and PPR set, respectively.

Investigators also noted that pharmacokinetic parameters were within the expected ranges. The mean VWF;RCo half-life was 11.7 hours. All 45 bleeds treated with pdVWF/FVIII during the prophylaxis period in 10/12 patients in the FAS group were managed successfully. Epistaxis was the most common bleed type across patients, while only 1 joint bleed was observed during the study. Additionally, most bleeds (95.6%) only required 1 infusion.

“I’m really hopeful that our body of evidence will help impact research for the next 5-10 years to come, and we certainly plan on building upon the work we have done by increasing the cohort size and having a more detailed long-term safety profile in the real world,” Jain said. “Hopefully, these data will be shared at the next meeting.”

Editor's Note: Jain reports disclosures with Hemex Health, Sanofi, NovoNordisk, Blue Bird Bio, and Beam Therapeutics.

Jain A, Leissinger C, Vdovin V, et al. Plasma-derived VWF/FVIII prophylaxis in children under 6 with VWD: First results from wil-33. Abstract presented at the 67th American Society of Hematology Annual Meeting. Orlando, FL. December 6-9, 2025.

Sidonio RF Jr, Boban A, Dubey L, et al. von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease. Blood Adv. 2024;8(6):1405-1414. 

Videos

Jain describes the phase 3 WIL-33 trial, which marked the first test of pdVWF/FVIII in adolescent patients aged <6 years.

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

Abigail Brooks is an assistant managing 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.

The US Food and Drug Administration has approved Amgen’s inebilizumab-cdon (Uplizna) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive, offering patients a new targeted treatment option that has the potential for long-term disease control with just 2 doses a year after 2 initial loading doses.

As described in a December 11, 2025, release from the Company, the approval marks inebilizumab-cdon’s third indication and is supported by data from the Myasthenia Gravis Inebilizumab Trial (MINT), the largest phase 3 biologic study to include both AChR+ and MuSK+ patients, and the first to successfully incorporate a steroid taper into its protocol.

"Managing a rare and chronic illness can mean facing unpredictable relapsing symptoms and demanding treatment schedules," Samantha Masterson, president and chief executive officer of the Myasthenia Gravis Foundation of America, said in a statement.

 "This approval marks an important milestone, offering durable efficacy and a dosing schedule that provides people living with generalized myasthenia gravis 6 months of treatment-free time between maintenance doses."

Inebilizumab-cdon is a humanized monoclonal antibody (mAb) that causes targeted and sustained depletion of key cells that contribute to underlying disease process (autoantibody-producing CD19+ B cells, including plasmablasts and some plasma cells). It has previously been approved by the FDA for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD) in June 2020, and for the treatment of adult patients with Immunoglobulin G4-related disease (IgG4-RD) in April 2025.

MINT is a randomized, double-blind, placebo-controlled, parallel-group trial designed to evaluate the efficacy and safety of inebilizumab-cdon in adults with gMG. The trial enrolled 238 adults with gMG, including 190 patients who are AChR+ and 48 patients who are MuSK+.

The primary endpoint was change from baseline in MG-ADL score at week 26 in the combined study population. Key secondary endpoints included change from baseline in QMG scores in the combined study population; change from baseline in MG-ADL score at week 26 for the AChR+ cohort and separately the MuSK+ cohort; and change from baseline in QMG score at Week 26 for the AChR+ cohort and separately the MuSK+ cohort. MINT also includes an optional 3-year open-label treatment period.

In MINT, at week 26, inebilizumab-cdon demonstrated a 1.9-point difference in the MG-ADL score compared with placebo (-4.2 vs -2.2; 

<.0001). Benefits in the AChR+ patient subgroup continued through week 52, the longest randomized-controlled period for a phase 3 trial in gMG, with an exploratory analysis of AChR+ patients showing a 2.8-point difference in MG-ADL for inebilizumab-cdon compared with placebo (-4.7 vs -1.9; 95% CI, −3.9 to −1.7).

"[Inebilizumab-cdon] showed strong efficacy at 26 weeks in both AChR+ and MuSK+ patients, with AChR+ patients continuing to improve through 52 weeks in MINT," said Richard Nowak, MD, MS, global principal investigator and director of the Myasthenia Gravis Clinic at Yale University.

 "MINT also uniquely required steroid tapering, recognizing that long-term steroid use adds to the overall burden of disease. This approval brings a new first-in-class approach to gMG, expanding treatment options for clinicians and patients."

Amgen. FDA APPROVES UPLIZNA® FOR ADULTS WITH GENERALIZED MYASTHENIA GRAVIS. December 11, 2025. Accessed December 12, 2025. 

https://www.amgen.com/newsroom/press-releases/2025/12/fda-approves-uplizna-for-adults-with-generalized-myasthenia-gravis

Nowak RJ, Benatar M, Ciafaloni M, et al. A Phase 3 Trial of Inebilizumab in Generalized Myasthenia Gravis. 

Articles

The FDA has approved Amgen's inebilizumab-cdon for treating generalized myasthenia gravis (gMG) in adults with specific antibodies, marking its third indication. This approval is based on the Myasthenia Gravis Inebilizumab Trial (MINT), the largest phase 3 study for AChR+ and MuSK+ patients, which demonstrated significant efficacy and safety. Inebilizumab-cdon, a humanized monoclonal antibody, offers a novel treatment approach with a biannual dosing schedule. The trial showed significant improvements in MG-ADL scores, particularly in AChR+ patients, and incorporated a steroid tapering protocol.

The approval makes inebilizumab-cdon the first and only CD19-targeted B cell therapy indicated for anti-AChR and anti-MuSK Ab+ generalized myasthenia gravis.

FDA News

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.

FDA Approves Inebilizumab-cdon (Uplizna) for Generalized Myasthenia Gravis

Chelsie Derman is an associate editor for 

who covers psychiatry, sleep medicine, geriatrics, migraine, and allergy. 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.

Credit: The US Food & Drug Administration 

BioCryst announced on Friday, December 12, 2025, that the 

 approved berotralstat (ORLADEYO) oral granules for patients with 

This follows berotralstat’s 2020 FDA approval for the prevention of HAE attacks in adults and children aged ≥ 12 years.

“Pediatric patients living with HAE can experience significant burdens, not only from the disease itself but also from its treatments, affecting their physical, psychosocial, and developmental well-being,” said Raffi Tachdjian, MD, associate clinical professor of medicine & pediatrics at the David Geffen School of Medicine, University of California Los Angeles. “This new oral pellet formulation of ORLADEYO provides a more easily administered long-term-prophylactic option to help children with HAE better manage their disease, especially when the experience of infusions or injections is not ideal.”

This past year the FDA approved many treatments for HAE—

garadacimab-gxii (Andembry) in June, sebetralstat (Ekterly) in July, and donidalorsen (Dawnzera) in August

 Numerous experts said that, despite these approvals, a critical HAE care gap was the limited treatment options for children under 12 years.

“We are excited to make ORLADEYO oral pellets available to children living with HAE. The use of prophylactic therapy has been limited among younger children compared to adults, in part because of the lack of appropriate, less burdensome treatment options. ORLADEYO oral pellets have the potential to change how a new generation of children with HAE and their caregivers manage this condition, giving them more freedom to live a normal life,” said Charlie Gayer, president and chief commercial officer of BioCryst.

Until now, children under 12 with HAE could only take Berinert (plasma-derived esterase inhibitor (human)), Cinryze (C1 esterase inhibitor (human)), haegarda (c1 esterase inhibitor subcutaneous (human)), and lanadelumab-flyo (Takhzyro).

 Of these previously approved treatments, only Berinert and lanadelumab-flyo are approved to target HAE attacks in children as young as 2 years old.

 This berotralstat approval adds another treatment option for children experiencing HAE attacks.

Berotralstat, a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein, is the first oral therapy designed specifically to prevent attacks of HAE.

 Taking 1 capsule a day can prevent attacks by reducing the activity of plasma kallikrein.

The pediatric approval was based on positive interim data from APeX-P, the largest clinical trial evaluating prophylactic therapy for HAE in 29 patients aged 2 to 11 years (48.3% females; mean age, 8 years, mean HAE onset age, 2 years).

 The study, led by Jolanta Bernatoniene, MD, PhD, from Bristol Royal Hospital for Children in the UK, showed berotralstat provided early and sustained reductions in monthly attack rates. At a steady state, berotralstat reached peak concentration at a median of 3.9 hours (range, 0.9 – 6.0 hours).

 Investigators noted an increase in QT prolongation at dosages over the recommended 150 mg daily dose.

Berotralstat was well tolerated and had a consistent safety profile in children as compared to adults. Common adverse events included abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

“As we mark 5 years since ORLADEYO first transformed care for those with HAE ages 12 and older, this approval extends the benefits of oral prophylactic therapy to a vulnerable and important part of the HAE community, children ages 2 to less than 12. We couldn’t be more excited to bring this treatment option to these kids and their caregivers,” said Jon Stonehouse, chief executive officer of BioCryst. “ORLADEYO has been prescribed to more than 3,500 patients in the U.S. to date, and we’re honored to now bring an oral pellet formulation to children and their caregivers, answering the community’s heartfelt call for a long-term prophylactic treatment option that meets the unique needs of children."

BioCryst Announces FDA Approval of ORLADEYO® (berotralstat) Oral Pellets, First and Only Oral Prophylactic Treatment for Patients with HAE Aged 2 to <12 Years. BioCryst. Published December 12, 2025. Accessed December 12, 2025.

 https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-fda-approval-orladeyor-berotralstat-oral 

Kunzmann K. FDA Approves Berotralstat as First Oral Hereditary Angioedema Prophylaxis. Hcplive. Published December 4, 2020. Accessed December 9, 2025. 

https://www.hcplive.com/view/fda-approves-berotralstat-oral-hereditary-angioedema-prophylaxis

Soteres D, Tachdjian R, Manning M. Rare Disease, Rapid Progress: FDA's HAE Approvals in 2025 Signal Changing Course. HCPLive. Published December 3, 2025. Accessed 9, 2025. 

https://www.hcplive.com/view/rare-disease-rapid-progress-fda-hae-approvals-in-2025-signal-changing-course

US Hereditary Angioedema Association. Haea.org. Published 2020. 

FDA Expands Lanadelumab for Preventing HAE Attacks in Younger Children. HCPLive. Published February 4, 2023. Accessed 9, 2025. 

https://www.hcplive.com/view/fda-expands-lanadelumab-preventing-hae-attacks-younger-children

U.S. FDA Approves CSL Behring’s Berinert® as the First and Only Pediatric Treatment for Hereditary Angioedema (HAE). Global Newsroom | CSL. Published July 18, 2016. Accessed December 9, 2025. 

https://newsroom.csl.com/2016-07-18-U-S-FDA-Approves-CSL-Behrings-Berinert-R-as-the-First-and-Only-Pediatric-Treatment-for-Hereditary-Angioedema-HAE 

Derman C. FDA Accepts BioCryst’s NDA for Berotralstat Oral Granules in Children With HAE. HCPLive. Published May 14, 2025. Accessed December 9, 2025. 

https://www.hcplive.com/view/fda-accepts-biocryst-s-nda-for-berotralstat-oral-granules-in-children-with-hae

Derman C. Oral Berotralstat Shows Strong Safety, Efficacy in Young Children with HAE. HCPLive. Published August 5, 2025. Accessed December 9, 2025. 

https://www.hcplive.com/view/oral-berotralstat-shows-strong-safety-efficacy-in-young-children-with-hae

The FDA has approved berotralstat (ORLADEYO) oral granules for children aged 2-11 with hereditary angioedema (HAE), expanding its 2020 approval for those aged 12 and older. This new formulation offers a less burdensome prophylactic option compared to infusions or injections, addressing a critical gap in pediatric HAE care. The approval is based on positive interim data from the APeX-P trial, which demonstrated significant reductions in attack rates. Berotralstat is well-tolerated in children, with common adverse events including abdominal pain and vomiting.

Berotralstat (ORLADEYO) oral granules become the first FDA-approved therapy specifically designed to prevent HAE attacks in children aged 2 – 11 years.

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.

FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE  

The US Food and Drug Administration has approved Acadia Pharmaceuticals’ trofinetide (Daybue Stix) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients ≥ 2 years of age.

As described in a December 12, 2025, release from the Company, the new formulation is expected to deliver the same efficacy and safety profile of trofinetide oral solution, while offering children and adults living with Rett syndrome new flexibility and choice regarding the dose volume and taste of their trofinetide treatment.

“The new formulation gives us an additional option for treatment with DAYBUE, allowing us to better customize care for our patients,” said Jennifer Martelle Tu, MD, PhD, Director of Katie's Clinic for Rett Syndrome and Associate Professor of Neurology, UCSF Benioff Children's Hospitals, Oakland. “DAYBUE STIX is a powder for oral solution that caregivers can mix with a variety of water-based liquids providing flexibility to modify the taste and volume of their loved-one's dose. We know that this kind of adaptability is something many Rett families have been seeking.”

Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. It became 

the only FDA-approved treatment for Rett syndrome in 2023. 

The mechanism by which it exerts therapeutic effects in patients with Rett syndrome is unknown, but in animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.

“DAYBUE, the first and only approved treatment for Rett syndrome, has provided thousands of patients an important therapeutic option,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “With the approval of DAYBUE STIX, we are building on our commitment to turn scientific promise into meaningful innovation by introducing a new formulation informed by invaluable feedback from patients, caregivers and healthcare providers.”

The efficacy and safety of this new formulation was assessed in the LAVENDER™ study in patients with Rett syndrome. The approval was informed by the results of a bioequivalence study, which demonstrated that both original trofinetide oral solution and the new trofinetide for oral solution powder formulation provide comparable exposure. This confirmed bioequivalence means patients can expect the same efficacy and safety established by the oral solution formulation when using trofinetide for oral solution powder formulation.

As described in the release from Acadia, DAYBUE STIX will be available on a limited basis starting in the first quarter of 2026 and more broadly early in the second quarter of 2026, and the current oral solution formulation will remain available.

Acadia Pharmaceuticals. Acadia Pharmaceuticals Announces FDA Approval of DAYBUE® STIX (trofinetide) for Oral Solution, a New Powder Formulation of Trofinetide for the Treatment of Rett Syndrome. December 12, 2025. Accessed December 12, 2025. 

https://acadia.com/en-us/media/news-releases/acadia-pharmaceuticals-announces-fda-approval-daybuer-stix

FDA. FDA approves first treatment for Rett Syndrome. March 13, 2023. Accessed December 12, 2025. 

https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-rett-syndrome

The FDA has approved Acadia Pharmaceuticals' trofinetide (Daybue Stix) for oral solution, a new powder formulation for treating Rett syndrome in patients aged 2 and older. This formulation offers flexibility in dose volume and taste, enhancing treatment customization. Trofinetide, a synthetic analog of insulin-like growth factor 1, is the only FDA-approved treatment for Rett syndrome. The new formulation's efficacy and safety were confirmed through bioequivalence studies, ensuring comparable exposure to the original oral solution. Daybue Stix will be available in early 2026, alongside the existing oral solution.

The approval of a dye- and preservative-free powder formulation of trofinetide offers a new option for adult and pediatric patients with Rett syndrome.

FDA Approves New Powder Formulation of Trofinetide (Daybue) for Rett Syndrome

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for HCPLive. He is also the executive producer of two podcasts: Skin of Color Savvy and The Medical Sisterhood. 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.

Apremilast is effective as an oral systemic therapy for 

, new data suggest, with consistent clinical efficacy, symptoms improvements, and quality-of-life benefits.

These findings from the phase 3 DISCREET study (NCT03777436) followed 32 weeks of analysis of this drug’s impact on genital psoriasis. Apremilast is an oral phosphodiesterase-4 (PDE4)-inhibitor approved for patients living with psoriasis. The medication previously demonstrated safety and efficacy in treating genital psoriasis following the 16-week results of DISCREET.

This new 32-week analysis, authored by investigators such as Joseph F. Merola, MD, MMSc, of UT Southwestern Medical Center, explored the twice-daily use of apremilast in the same patient group following the primary findings released at the 16-week mark.

“Product labelling in the United States has been revised to include these data supporting the efficacy of apremilast in genital psoriasis,” Merola et al wrote.

 “We report 32-week safety and efficacy results as well as post-hoc analyses of efficacy results.”

In their analysis, Merola and coauthors described the design of the DISCREET trial as randomized, double-blind, and placebo-controlled. The investigators' research was conducted across multiple centers located in Canada, Belgium, France, Italy, the US, and Germany. While details of DISCREET's design and methodology were previously highlighted, the team noted participants were assigned in a 1:1 ratio to be treated either with apremilast or placebo through a centralized Interactive Response Technology system via permuted block randomization.

Adults with moderate to severe genital psoriasis, as indicated by a baseline modified static Physician’s Global Assessment of Genitalia (genital PGA or sPGA-G) score of 3 or higher, were deemed as eligible to take part in the trial. All of those involved as participants had either insufficient response to or intolerance of topical drugs. Additionally, these individuals would also have had moderate to severe chronic plaque psoriasis for at least half a year, defined as an overall static Physician’s Global Assessment (sPGA) score of 3 at least, and non-genital plaque psoriasis involving at least 1% of patients' body surface area (BSA).

In terms of treatment Merola et al administered apremilast 30 mg, taken orally, on a twice-daily basis or provided participants with a matched placebo for 16 weeks during the placebo-controlled phase of DISCREET. This was then followed by a 16-week extension phase, during which the investigators provided all subjects with apremilast. Individuals who had been on placebo switched to active therapy from Weeks 16 - 32 (placebo/apremilast cohort). At the same time, individuals originally assigned to the apremilast cohort continued treatment for the full 32 weeks (apremilast/apremilast group).

At screening, at baseline, and at the 2, 4, 8, 12, 16, 20, 24, and 32-week marks, study visits took place. Merola and colleagues' follow-up interaction took place 4 weeks following the last apremilast dose. There were 289 patients who were randomized, with 229 entering the study's apremilast extension period. Among these subjects, 110 were transitioned by Merola and coauthors from placebo to apremilast at the 16-week mark. 119 remained on continuous apremilast throughout.

By Week 32, the investigative team found attainment of a modified genital PGA response, defined as a score of 0 or 1 with a reduction of at least 2 points from baseline, was seen among 51.8% of those included in the placebo/apremilast group (95% CI: 42.6–60.9).

 It was also seen among 40.3% of the apremilast/apremilast group (95% CI: 32.0–49.3).

By the conclusion of the study's 32-week period, the team identified improvements in overall skin disease, genital psoriasis signs and symptoms, and quality-of-life measures. They also found these results to be comparable between subjects who initiated apremilast at randomization and those who began treatment at the 16-week mark.

Additionally, the Merola et al's safety findings revealed the most commonly reported treatment-emergent adverse events during apremilast exposure to be instances of diarrhea (25.4%), nausea (19.4%), and headaches (17.9%).

“Based on the consistent clinical efficacy, improvements in symptoms and QoL benefit seen in DISCREET, apremilast appears to be an effective oral therapy in patients with moderate to severe genital psoriasis,” the investigators wrote.

Merola JF, Guenther L, Yamauchi P, et al. Results from the 32-week, phase 3 DISCREET study of apremilast in patients with moderate to severe genital psoriasis. J Eur Acad Dermatol Venereol. 2025; 00: 1–11. 

Merola JF, Parish LC, Papp KA, et al. Efficacy and safety of apremilast in patients with moderate-to-severe genital psoriasis: Results from DISCREET, a phase 3 randomized, double-blind, placebo-controlled trial. J Am Acad Dermatol. 2024 Mar;90(3):485-493. 

Apremilast, an oral PDE4 inhibitor, has shown efficacy in treating moderate to severe genital psoriasis, as evidenced by the 32-week phase 3 DISCREET study. The study demonstrated significant clinical efficacy, symptom improvement, and quality-of-life benefits. Conducted across multiple centers, the trial involved a randomized, double-blind, placebo-controlled design. Participants initially on placebo switched to apremilast after 16 weeks, with notable improvements observed by week 32. Common adverse events included diarrhea, nausea, and headaches. These findings support apremilast as an effective oral therapy for genital psoriasis.

The phase 3 DISCREET study explored the safety and efficacy of apremilast in treating patients with moderate to severe genital psoriasis over 32 weeks.

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.

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.

Apremilast Effective Treatment for Moderate to Severe Genital Psoriasis at 32 Weeks

2025 data show the US is still failing to adequately identify and initiate care for deadly lung disease. Experts discuss what it will take to turn the tide on this public health issue.

This Year In Medicine

Pulmonology

Infectious Disease condition center page is a comprehensive resource for clinical news and insights on respiratory health. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for asthma, COPD, interstitial lung disease, COVID-19, and more.

COPD

COPD condition center page, resources on the topics of medical news and expert insight into chronic obstructive pulmonary disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on COPD research, treatment, and drug development.

Lung Cancer

Getting Through the Door: How COPD, Lung Cancer Screening Remains Minimal

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.

Weight-adjusted waist index outperforms obesity indices for cardiovascular disease prediction in cardiovascular-kidney-metabolic syndrome 


Nephrology

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

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...

Weight-Adjusted Waist Index Can Predict CVD Risk in Cardiovascular Kidney Metabolic Syndrome


What are your impressions of the Phase 2b data of zasocitinib in psoriasis, particularly in terms of Psoriasis Area and Severity Index (PASI) responses, dose-dependent effects, and tolerability? How do you see these results influencing your real-world patient selection?

Efficacy and Safety of Zasocitinib in Psoriasis and the Ideal Candidates for TYK2 Inhibitors

The panel reviews the HELIOS-B Phase 3 trial evaluating vutrisiran, a transthyretin silencer, in patients with ATTR-CM. Participants were randomized to receive vutrisiran or placebo, with approximately 40% already taking tafamidis at trial entry, particularly in the United States. The study was designed with predefined primary and secondary endpoints, assessing outcomes both in the overall population and in those not on baseline tafamidis. The trial demonstrated statistically significant results across all major endpoints, including reductions in all-cause mortality and cardiovascular hospitalizations, with notable benefit observed both in monotherapy and combination-therapy subgroups.
The panel highlights the magnitude of treatment effect and notes that these findings supported FDA approval of vutrisiran for ATTR-CM. They also emphasize its favorable safety profile, explaining that common adverse events—such as cardiac failure and arrhythmias—reflect the underlying disease rather than the drug itself. Overall, the experts describe vutrisiran as a meaningful and well-tolerated therapeutic advancement for patients with ATTR-CM.


Video Series

Evaluating Efficacy and Safety of Vutrisiran in ATTR-CM from the HELIOS-B Trial

The panelists review EADV 2025 data evaluating whether bimekizumab may help prevent PsA development in patients with psoriasis only. The study followed psoriasis patients prospectively and stratified risk using clinical factors and a patient questionnaire (PASE), treating scores <47 as low risk and ≥47 as high risk. Among patients with >1 risk factor treated with bimekizumab, over 96% maintained PASE scores <47 on longitudinal follow-up—used here as a patient-reported surrogate for not developing PsA symptoms (noting the limitation of no comparator arm). These findings align with real-world practice that prioritizes identifying high-risk patients and monitoring symptom trajectories over time. Overall, the experts interpret the stratified results as supportive—though preliminary—evidence that sustained IL-17A/F blockade may reduce transition to PsA in higher-risk psoriasis populations, while emphasizing the need for prospective, comparative data.

Bimekizumab in Psoriasis: EADV 2025 Insights and Approaches to Patient Monitoring

The panel discusses the concept of achieving a rapid reduction, or “knockdown,” of transthyretin (TTR) production in ATTR-CM. They note that the rationale is intuitive: slowing or halting new amyloid formation as early as possible may help interrupt disease progression. The comparison is made to AL amyloidosis, where shutting down the source of the abnormal protein is central to treatment. However, the panel emphasizes that ATTR-CM develops gradually over many years, and as a result, even when TTR production is reduced, clinical improvement is typically gradual rather than immediate.
The experts highlight the importance of setting realistic patient expectations—treatment reduces ongoing injury but does not rapidly remove existing amyloid deposits. They also discuss the need to understand which symptoms are truly amyloid-related versus coincidental, and they stress the long-term, supportive relationships clinicians build with patients and families, particularly when helping individuals adjust goals and maintain quality of life over time.


Clinical Implications of Rapid Transthyretin Knockdown in ATTR-CM

In this episode, the panelists share clinical perspectives on positioning IL-17 inhibitors within psoriasis treatment. The experts discuss that among available biologic options, IL-17 inhibitors provide rapid and durable skin clearance, making them an important choice for patients with moderate to severe psoriasis. They highlight the significance of dual IL-17A and IL-17F inhibition, noting that it offers greater depth of response compared to targeting IL-17A alone. When selecting therapy, the panel emphasizes tailoring treatment to individual patient factors—such as nail involvement, disease burden, or potential psoriatic arthritis risk. They also consider head-to-head data showing that IL-17 inhibitors can achieve superior outcomes compared with other classes, including IL-23 inhibitors, in certain patient groups. The experts conclude that positioning IL-17 inhibitors early in the treatment algorithm may not only improve skin outcomes but also support prevention of disease progression.

Clinical Perspectives for Positioning IL-17 Inhibitors in Psoriasis Care

Multicenter study shows patients with both CKD and gout achieve better urate control on febuxostat than those with gout alone, despite the lower doses.


Rheumatology

Rheumatology condition center page is a comprehensive resource for clinical news and insights on rheumatologic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for arthritis, gout, nr-AxSpA, and more.

Gout

On the HCPLive gout page, resources on the topics of medical news and expert insight into gouty arthritis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on gout, and more.

Chronic Kidney Disease

Febuxostat Lowers Serum Uric Acid More Effectively in Gout Patients With CKD

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Understanding the FDA-Approved TYK2 Inhibitor in Psoriasis

Historic progress in renal xenotransplantation in 2025 brings pig kidney transplants closer to clinical reality amid an ongoing donor organ shortage.

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