HCPLive Year in Review: Top 10 FDA Approvals for 2024

Published on: December 31, 2024

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

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.

Most Popular FDA Approvals of 2024

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.

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.

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.

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.

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.

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.

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.

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.

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.