HCPLive Editorial Staff

HCPLive Year in Review: Top 10 FDA Approvals for 2024

2024 has been a year of remarkable innovation and progress in medicine, with the US Food and Drug Administration granting approvals that have captivated both clinicians and patients. From cutting-edge therapies addressing rare diseases to new standards of care in widespread conditions, the medical community has witnessed a surge of interest in advancements that have reshaped treatment landscapes.

To highlight the year’s most impactful developments, we have compiled a list of the 10 most-viewed FDA approval articles on HCPLive. These rankings reflect the treatments and breakthroughs that resonated most with our audience, showcasing the priorities and interests of healthcare professionals in 2024. This list also features links to related coverage with expert perspectives from our coverage and that of our sister publications, within the HCPLive Network and MJH Life Sciences family.

Semaglutide (Wegovy) Receives FDA Label Expansion to Include Cardiovascular Risk Reduction

On March 8, 2024, the FDA approved an expanded label indication for semaglutide 2.4 mg (Wegovy) to reduce the risk of cardiovascular death, heart attack, and stroke in adults with cardiovascular disease and obesity or overweight. This approval marks the first time a weight loss medication has been approved for cardiovascular risk reduction in this patient population.

The approval is based on data from the SELECT trial, a 17,604-patient study that demonstrated a 20% relative reduction in the risk of the primary cardiovascular events (HR, 0.80; P <.001) in patients receiving semaglutide compared to placebo. Additionally, semaglutide was associated with a mean body weight reduction of 9.39% versus 0.88% for placebo. Further analysis also suggested trends towards benefits in reducing cardiovascular death, heart failure hospitalization, and all-cause mortality.

Diabetes Dialogue: SELECT, Semaglutide, & the Future of Incretin Therapies, with A. Michael Lincoff, MD

FDA Approves Dupilumab for EoE in Children Aged 1 to 11 years

On January 15, 2024, the FDA approved dupilumab (Dupixent; Regeneron and Sanofi) for eosinophilic esophagitis (EoE) in children aged 1–11 years weighing at least 33 pounds (15 kg), the first treatment for this age group. The approval, under Priority Review, expands on its prior indication for patients aged 12 years and older.

Phase 3 EoE KIDS trial data showed 66% of children on higher dose dupilumab achieved histological remission versus 3% on placebo, with 53% maintaining remission at 52 weeks. Improvements in symptoms, endoscopic findings, and quality of life were also reported.

"Having another treatment option can significantly improve families' quality of life," said Theresa Bingemann, MD, associate professor of pediatrics and medicine as well as the program director of Allergy and Immunology Fellowship at the University of Rochester School of Medicine and Dentistry.

Long Time Coming: Pediatric Gastroenterologists React to New Dupilumab EoE Approval

FDA Approves Ensifentrine (Ohtuvayre) for COPD

On June 26, 2024, the FDA approved ensifentrine (Ohtuvayre) for the treatment of chronic obstructive pulmonary disease (COPD). Developed by Verona Pharma, ensifentrine is a first-in-class dual inhibitor of the phosphodiesterase 3 and 4 (PDE3 and PDE4) pathways, marking the first novel mechanism of action approved for COPD in a decade.

The approval is based on the results of the phase 3 ENHANCE clinical trial program, which included approximately 800 patients with moderate to severe COPD. The trials demonstrated that ensifentrine significantly improved lung function, with a 94 mL change in forced expiratory volume (FEV1) AUC at 12 weeks (P <.0001). Patients also experienced a 42% reduction in COPD exacerbations compared to placebo (P = .0109). Additionally, ensifentrine improved health-related quality of life, with positive effects on St. George's Respiratory Questionnaire and Evaluating Respiratory Symptoms scores.

This approval was the first of 2 significant COPD approvals in 2024, with dupilumab earning a nod from the FDA as an add-on treatment in patients with uncontrolled COPD.

Expert Perspectives: What the Ensifentrine FDA Approval Means for COPD

Resmetirom (Rezdiffra) Receives Historic FDA Approval for Noncirrhotic NASH

On March 14, 2024, the FDA granted accelerated approval to resmetirom (Rezdiffra) for the treatment of noncirrhotic nonalcoholic steatohepatitis (NASH) with moderate to advanced fibrosis, making it the first approved treatment for this progressive liver disease. This approval follows the completion of 18 clinical studies, including phase 2 and 3 trials, and is based on the ongoing phase 3 MAESTRO-NASH trial, which demonstrated resmetirom's ability to improve liver fibrosis and resolve NASH without worsening fibrosis.

In the MAESTRO-NASH trial, 25.9% of patients receiving resmetirom 80 mg and 29.9% of those receiving 100 mg achieved NASH resolution without worsening fibrosis, compared to 9.7% in the placebo group. Additionally, resmetirom showed a reduction in LDL cholesterol by up to 16.3%. The safety profile was favorable, with most adverse events being mild or moderate in severity.

Expert Perspectives: Understanding Resmetirom's Potential Based on MAESTRO-NASH

FDA Approves Donanemab for Early Symptomatic Alzheimer Disease

On July 2, 2024, the FDA approved Eli Lilly's donanemab (Kisunla), a monthly IV infusion, for early symptomatic Alzheimer disease, including mild cognitive impairment and mild dementia stages with confirmed amyloid pathology. Donanemab is the first antiamyloid therapy with evidence supporting cessation of treatment upon amyloid plaque clearance, potentially lowering costs and infusion burdens.

The approval was supported by results from the phase 3 TRAILBLAZER-ALZ-2 trial, where donanemab demonstrated a significant slowing of cognitive and functional decline compared with placebo, particularly in patients with low/medium tau pathology (35.1% disease progression slowing). The study also showed substantial amyloid clearance, with 80.1% of treated patients achieving plaque removal by week 76.

Safety data revealed a 24% incidence of ARIA-E, predominantly mild to moderate, with most cases resolving within 10 weeks. Donanemab outperformed aducanumab in amyloid clearance in a head-to-head trial, emphasizing its potential in evolving the standard of care for Alzheimer disease.

Mind Moments: Special Episode with Joel Salinas, MD, MBA

FDA Approves Tirzepatide for Obstructive Sleep Apnea in Obesity

On December 20, 2024, Eli Lilly announced the FDA approval of tirzepatide (Zepbound) for adults with moderate-to-severe obstructive sleep apnea (OSA) and obesity. This dual GIP/GLP-1 agonist demonstrated up to 20% weight loss and a reduction of ≥25 breathing interruptions per hour during sleep in the SURMOUNT-OSA trial.

In patients without positive airway pressure (PAP) therapy, tirzepatide reduced breathing disruptions by 25 hourly events compared to 5 for placebo, with similar outcomes in those using PAP therapy. Nearly 50% of tirzepatide-treated patients achieved remission or mild, non-symptomatic OSA.

Safety data aligned with previous trials, showing mild-to-moderate gastrointestinal events. Lilly emphasized tirzepatide should be used alongside a reduced-calorie diet and increased physical activity to address OSA and obesity. "This approval offers a transformative treatment option for patients," stated Patrik Jonsson, Lilly’s executive vice president.

Atul Malhotra, MD: Tirzepatide for Obstructive Sleep Apnea and Obesity

FDA Approves Xanomeline and Trospium Chloride (Cobenfy) for Schizophrenia

On September 26, 2024, the FDA approved xanomeline and trospium chloride capsules (Cobenfy) for schizophrenia, marking the first new pharmacological approach for the condition since the 1970s. Developed by Bristol-Myers Squibb, xanomeline and trospium chloride capsules are the first antipsychotic targeting cholinergic rather than dopamine receptors, providing a novel mechanism of action.

Approval was based on the phase 3 EMERGENT trials, which demonstrated significant reductions in PANSS total scores compared with placebo (-20.6 vs -12.2; LS mean difference, -8.4; P < .001) and long-term safety, with 75% achieving ≥30% symptom improvement at 52 weeks. Unlike traditional antipsychotics, xanomeline and trospium chloride capsules avoids side effects such as weight gain, somnolence, and movement disorders.

“This approval offers a new alternative to the antipsychotic medications previously prescribed for schizophrenia,” said Tiffany Farchione, MD, of the FDA’s Center for Drug Evaluation and Research.

Expert Perspectives: Applying EMERGENT Trial Results to Real-World Clinical Practice for Schizophrenia

FDA Approves Acoramidis (Attruby) for ATTR-CM

On November 22, 2024, the FDA approved acoramidis (Attruby), an orally administered stabilizer of transthyretin (TTR), for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM). This approval marks acoramidis as the first and only treatment with a label specifying near-complete TTR stabilization.

The approval follows data from the phase 3 ATTRibute-CM trial, a double-blind, placebo-controlled study with 632 patients, which showed significant reductions in all-cause mortality and cardiovascular-related hospitalizations. The trial demonstrated that acoramidis reduced composite all-cause mortality and recurrent cardiovascular-related hospitalization by 42% compared to placebo at 30 months. Additionally, acoramidis showed a 50% reduction in the cumulative frequency of cardiovascular-related hospitalizations.

“With continued advances in therapy, this previously fatal disease is becoming a manageable chronic cardiovascular condition,” said Martha Grogan, MD, associate professor of medicine at the Mayo Clinic.

Recent Advances Have Improved Prognosis in ATTR-CM Patients, with Ahmad Masri, MD, MS

FDA Approves Pfizer’s RSV Vaccine for Expanded Population of Adults Aged 18 to 59 Years

On October 24, 2024, Pfizer announced FDA approval of its bivalent RSV prefusion F vaccine (Abrysvo) for preventing lower respiratory tract disease (LRTD) caused by RSV in adults aged 18–59 at increased risk due to chronic conditions. This expands the vaccine's indication beyond adults 60+ and pregnant women, making it the broadest indication for an adult RSV vaccine.

Approval was based on phase 3 MONeT trial data, which demonstrated strong immune responses and tolerability among high-risk individuals with chronic conditions such as asthma, diabetes, and heart failure. The vaccine’s safety profile was consistent with earlier trials, with mild injection site pain, fatigue, and headache as the most common side effects. Pfizer plans to publish trial findings and noted the vaccine’s effectiveness in targeting RSV-A and RSV-B with a single dose, offering a vital tool for high-risk populations.

FDA Approves Topical Berdazimer Gel for Molluscum Contagiosum

On January 5, 2024, the FDA approved berdazimer gel (Zelsuvmi, 10.3%) for treating molluscum contagiosum in patients aged 1 year and older, announced by Ligand Pharmaceuticals, Inc. This marks the first topical drug for molluscum that can be applied at home by patients or caregivers.

The approval is based on phase 3 B-SIMPLE4 trial results, which showed significant improvements in lesion clearance after 12 weeks, with mild application site pain and erythema as the most common side effects. Molluscum contagiosum affects 6 million Americans annually, mainly children, often remaining untreated.

This approval adds to the treatment options for the viral skin infection, joining VP-102 (YCANTHE), a cantharidin-based therapy approved in July 2023.

Reviewing New Molluscum Therapies, with Nanette B. Silverberg, MD

Articles

The year 2024 has seen significant advancements in medicine, with the FDA approving numerous groundbreaking therapies. These include semaglutide for cardiovascular risk reduction in obesity, dupilumab for eosinophilic esophagitis in young children, and ensifentrine for COPD. Resmetirom received approval for noncirrhotic NASH, while donanemab was approved for early Alzheimer's disease. Other notable approvals include tirzepatide for obstructive sleep apnea, xanomeline and trospium chloride for schizophrenia, acoramidis for ATTR-CM, Pfizer's RSV vaccine for a broader adult population, and berdazimer gel for molluscum contagiosum.

To celebrate the close of 2024, we're recapping the 10 most popular approval articles across the HCPLive Network of brands.

As we turn the page on 2024, healthcare systems, professionals, and patients, in many ways, find themselves at a crossroads as they prepare to enter 2025.

2024 was a record year in medicine. With a slew of historic approvals in a multitude of disease states, many specialties find themselves amidst a renaissance of care.

However, even with all this advancement, for many, this year was more of the same—earmarked by struggles to overcome the impact of decades-long barriers to care to provide equitable access and care for underserved communities.

, the 8th edition of our annual series reflecting on the groundbreaking stories, persistent challenges, and transformative innovations in healthcare, will feature in-depth articles and multimedia content, spotlighting the most impactful developments within our areas of focus.

As always, this year’s series is a collaboration across the 

From new therapeutic advances in MASH/MASLD, heart failure, and menopause to the profound early impact of artificial intelligence in neurology, from regulatory milestones like needle-free treatments and home OCT to the evolving understanding of antimicrobial resistance and long COVID, this year’s stories reflect the diversity and dynamism of medicine in 2024.

 on Facebook, Twitter, and LinkedIn using the hashtag 

, or follow the series on HCPLive’s homepage and daily newsletter. Thank you for joining us in this exploration of healthcare’s continued evolution.

In the future, you will be able to find a list and summaries for each piece of content featured in our 

series. Remember to check back throughout December for new installments and insights!

Endoscopy in the Age of GLP-1 RAs: 2024 Sees New Risk-Based Perioperative Guidance

That was the question for many in 2024 as the community processed how to digest and implement new information on withholding GLP-1 RAs ahead of endoscopic procedures during 2024. 

System Integration: How AI Is Weaving Itself into Neurology

Artificial intelligence has quickly found a home in medicine, but what does it mean for neurology specifically? Find out in this article, with perspective across more than half a dozen disease states in neurology. 

A Pillared Approach: A Suite of Therapies for a Spectrum of Disease

In recent years, a collection of therapeutic classes and agents have redefined the management of metabolic disease. In 2024, these therapies entered a new realm—heart failure with preserved ejection fraction. 

The Long COVID Patient Journey: A Complex and Uncertain Road

More than 4 years removed from the peak of the COVID-19 pandemic in 2020, health systems are now grappling with challenges posed by long-term sequelae of the disease. 

Bringing It All Back Home: Reshaping Ophthalmology's Clinic with Home OCT

The FDA-approved Scanly, a home-based OCT device, revolutionizes nAMD care by empowering patients with self-monitoring and enhancing personalized treatment.

Where is the Mental Health Crisis Heading in 2025? Hear from Experts

America's mental health crisis continues to escalate—what drives it, and what lies ahead in 2025? Hear insight from psychiatry and psychology experts.

Diabetes Dialogue: Celebrating 2024 - The Year of the Continuous Glucose Monitor

In a special edition of HCPLive's flagship diabetes podcast, hosts reflect on the advancement in CGM that reshaped care for diabetes and overall health during 2024, which marked the 25th anniversary of the first FDA approval for a CGM system. 

2024’s Needle-Free Approvals Transform Management of Anaphylaxis, Influenza in Children

Needle-free treatments, such as neffy and FluMist, offer promising alternatives for children and adults with needle phobia, ensuring access to critical care.

The Slow-Moving Antimicrobial Resistance Train is Picking-up Morbidity and Mortality Steam

Resistance is expected to worsen significantly, but can a combination of both ground-level clinician stewardship actions as well as systemic reforms such as the Pasteur Act at least slow down the train?

Hope on the Horizon: 2 Food Allergy Breakthroughs in 2024

A trio of subject matter experts reflect on some of the year’s most important developments in the food allergy space, namely the approvals of omalizumab and neffy.

How Digital Health Tools, AI, and Wearables are Transforming Care and Self-Management

Adoption of telemedicine, wearables, and health apps surged during COVID-19. These technologies have become a permanent fixture in healthcare, offering convenience and reducing exposure risks.

Advancing the Diagnostic Criteria for Multiple Sclerosis: A New Era of Early Detection and Precision

With new revisions of the MS diagnostic criteria being made, clinicians highlighted key updates and the broader implications it has for diagnosis, treatment, and healthcare practices worldwide.

The Rise, Fall (and Future?) of MDMA-Assisted Therapy

MDMA-assisted therapy, a source of hope for many, went on a tumultuous journey in 2024, going from historic hype to setbacks.

Crisis Point: Benzene Concerns in Acne Care

In this episode of Crisis Point, David Light, Neal Bhatia, MD, and Christopher Bunick, MD, PhD, discuss this year's news regarding benzene found in acne products.

An overview and landing page for future installments from HCPLive's 8th annual This Year in Medicine Series. 

This Year in Medicine 2024: Stories that Defined Healthcare

Early identification of moderate-to-advanced fibrosis is essential for improving outcomes in patients with 

. Although liver biopsy has long been viewed as the “gold standard” for assessing fibrosis and steatosis, its clinical utility is limited by its cost, invasiveness, and risk of complications, underscoring the importance of noninvasive tests that overcome these limitations.

American Association for the Study of Liver Diseases (AASLD)

 released a pair of practice guidelines for the use of blood- and imaging-based noninvasive assessment of fibrosis and steatosis, both intended for adult and pediatric healthcare providers who see patients with chronic liver disease.

Test your knowledge of the 2024 AASLD Practice Guidelines on imaging-based and blood-based noninvasive liver disease assessment of hepatic fibrosis and steatosis with this HCPLive Clinical Quiz!

Eliminate the need for imaging modalities

Sterling RK, Duarte-Rojo A, Patel K, et al. AASLD Practice Guideline on imaging-based noninvasive liver disease assessment of hepatic fibrosis and steatosis. 

Sterling RK, Patel K, Duarte-Rojo A, et al. AASLD Practice Guideline on blood-based noninvasive liver disease assessment of hepatic fibrosis and steatosis. 

Early detection of moderate-to-advanced fibrosis is crucial for better outcomes in chronic liver disease patients. While liver biopsy is the traditional "gold standard" for assessing fibrosis and steatosis, its limitations include cost, invasiveness, and potential complications. This highlights the need for noninvasive alternatives. In 2024, the American Association for the Study of Liver Diseases (AASLD) introduced practice guidelines for noninvasive blood- and imaging-based assessments of fibrosis and steatosis, targeting both adult and pediatric healthcare providers managing chronic liver disease patients.

Test your knowledge of the 2024 AASLD Practice Guidelines on blood- and imaging-based noninvasive liver disease assessment with this clinical quiz!

Clinical Quiz: AASLD Guidelines for Noninvasive Testing in Chronic Liver Disease

In 2023, a multisociety Delphi process yielded updated nomenclature for what was then known as nonalcoholic fatty liver disease (NAFLD), a common 

 estimated to affect up to 25% of US adults and 30% of the total global population.

Recognizing the stigmatizing nature of the terminology and fundamental inaccuracies related to the natural history of the disease, multiple global hepatology and gastroenterology societies, patients and patient advocacy organizations, regulatory experts, and industry representatives set out to rectify these issues with the implementation of revised 

metabolic dysfunction-associated steatotic liver disease (MASLD)

 published Practice Guidance on the Clinical Assessment and Management of NAFLD. In a later commentary intended to serve as an accompanying article to this guidance, AASLD sought to clarify its clinical application in the context of the new nomenclature.

Test your knowledge of the 2023 AASLD NAFLD Practice Guidance and the implications of the new MASLD nomenclature with this clinical quiz!

All recommendations for NAFLD remain applicable to MASLD.

Only advanced fibrosis management is applicable.

The guidelines require liver biopsies for all MASLD cases.

MASLD coexisting with mild alcohol consumption.

Metabolic dysfunction and alcohol-associated steatotic liver disease.

Metabolic and advanced liver dysfunction.

Noninvasive tests like FIB-4 and vibration-controlled transient elastography.

Imaging studies without cardiometabolic considerations.

Brooks, A. From NAFLD to MASLD: 2023 Brings New Liver Disease Nomenclature. HCPLive. December 13, 2024. Accessed November 26, 2024. 

https://www.hcplive.com/view/from-nafld-to-masld-2023-new-liver-disease-nomenclature

Rinella ME, Neuschwander-Tetri BA, Siddiqui MS, et al. AASLD Practice Guidance on the clinical assessment and management of nonalcoholic fatty liver disease. 

Kanwal F, Neuschwander-Tetri BA, Loomba R, et al. Metabolic dysfunction–associated steatotic liver disease: Update and impact of new nomenclature on the American Association for the Study of Liver Diseases practice guidance on nonalcoholic fatty liver disease. 

In 2023, a multisociety Delphi process led to the renaming of nonalcoholic fatty liver disease (NAFLD) to metabolic dysfunction-associated steatotic liver disease (MASLD) to address stigmatization and inaccuracies. This change was supported by global hepatology and gastroenterology societies, patient advocacy groups, and industry experts. The American Association for the Study of Liver Diseases (AASLD) published guidance on NAFLD's clinical assessment and management, later clarifying its application under the new MASLD nomenclature. This shift aims to improve understanding and management of the disease.

Test your knowledge of the AASLD NAFLD Practice Guidance and its clinical application in light of the new MASLD nomenclature.