Credit: US Food and Drug Administration

The first half of 2024 has been nothing short of historic for the medical community. Earmarked by first-of-their-kind indications and mechanisms of action, patients and practitioners have become the recipients of an onslaught of approvals and clearances with clinically meaningful implications from the US Food and Drug Administration. During the first 6 months of 2024 alone, the FDA approved 20 novel agents, with indications ranging from rare disease to multiple forms of cancer to common metabolic health disorders.

To celebrate the first half of 2024, we have compiled a recap for each of the 20 novel drug approvals from January through June, including links to related coverage from HCPLive and other brands within the MJH Life Sciences family of brands, including OncLive, Dermatology Times, Ophthalmology Times, Pharmacy Times, and Contemporary OB/GYN.

Novel Drug Approvals Recap: First Half 2024

1. Berdazimer (Zelsuvmi)

Date: January 05, 2024
Indication: Molluscum Contagiosum
Background: Approval of berdazimer gel 10.3% was awarded to Ligand Pharmaceuticals based on the B-SIMPLE4 trial. The 891-patient, multicenter, vehicle-controlled, double-blind, phase 3 randomized clinical trial met its primary endpoint of complete clearance of molluscum contagiosum lesions at week 12 versus vehicle.
Related Coverage: FDA Approves Topical Berdazimer Gel for Molluscum Contagiosum

2. Cefepime/Enmetazobactam (Exblifep)

Date: February 22, 2024
Indication: Complicated Urinary Tract Infections
Background: Approval of cefepime/enmetazobactam was awarded to Allecra Therapeutics based on data from a phase 3 trial comparing it against piperacillin/tazobactam among 1041 patients. Results indicated a greater proportion of patients treated with cefepime/enmetazobactam achieved composite response defined as clinical cure and microbiological eradication at the test-of-cure visit on day 7 than those receiving piperacillin/tazobactam.
Related Coverage: FDA Approves Enmetazobactam for Treatment of cUTIs

3. LetibotulinumtoxinA-wlb (Letybo)

Date: February 29, 2024
Indication: Temporarily Improve the Appearance of Moderate-to-Severe Glabellar Lines
Background: Approval of letibotulinumtoxinA-wlb was awarded to Hugel Inc based on data from a trio of clinical trials referred to as the BLESS program. With a population of 1271 adults patients aged 18 to 75 years with moderate to severe glabellar lines, results indicated use was associated with a greater proportion of patients achieving achieving a score of 0 or 1 (none or mild) and at least a 2-grade improvement of wrinkles between the eyebrows at maximum frown from baseline to week 4 relative to placebo.
Related Coverage: Putting Education and Innovation at the Forefront at Winter Clinical Miami

4. Tislelizumab-jsgr (Tevimbra)

Date: March 13, 2024
Indication: Unresectable or Metastatic Esophageal Squamous Cell Carcinoma
Background: Approval of tislelizumab-jsgr was awarded to BeiGene based on the RATIONALE 302 study. A phase 3 trial of 562 patients with unresectable or metastatic esophageal squamous cell carcinoma with a history of prior systemic chemotherapy, results indicated use of tislelizumab significantly improved overall survival compared to chemotherapy.
Related Coverage: FDA Approves Tislelizumab for Advanced or Metastatic ESCC After Chemotherapy

5. Resmetirom (Rezdiffra)

Date: March 14, 2024
Indication: Noncirrhotic Non-Alcoholic Steatohepatitis (NASH) with Moderate to Advanced Liver Scarring
Background: Approval of resmetirom was awarded to Madrigal Pharmaceuticals based on the MAESTRO-NASH trial. The first FDA-approved medication to receive this indication, the pivotal trial of resmetirom concluded use was associated greater NASH resolution with no worsening of fibrosis among a population of 966 adult patients with NASH and fibrosis.
Related Coverage: HCPLive Special Report: How Approval of Resmetirom Impacts Management, Screening for NASH (Preview Below)

6. Aprocitentan (Tryvio)

Date: March 19, 2024
Indication: Hypertension
Background: Approval of aprocitentan was awarded to Idorsia Ltd based on the phase 3 PRECISION trial. The 730-patient trial concluded use of aprocitentan was associated with greater change in office systolic blood pressure than placebo therapy. The approval marked the first time the FDA had approved an oral antihypertensive agent with a new mechanism of action in more than 30 years.
Related Coverage: What to Know about the Aprocitentan Approval for Resistant Hypertension, with Michael Weber, MD (Preview Below)

7. Givinostat (Duvyzat)

Date: March 21, 2024
Indication: Duchenne Muscular Dystrophy (DMD) in Individuals Aged 6 years and Older
Background: Approval of givinostat was awarded to Italfarmaco SpA based on the EPIDYS trial. A 179-patient, phase 3 trial of male patients with DMD, the trial met its primary endpoint of change from baseline to month 18 in 4-stair climb (4SC) assessment time.
Related Coverage: FDA Approves Givinostat for Duchenne Muscular Dystrophy in Patients 6 Years and Up

8. Sotatercept-csrk (Winrevair)

Date: March 26, 2024
Indication: Pulmonary Arterial Hypertension
Background: Approval of sotatercept was awarded to Merck based on the STELLAR trial. A 323-patient, phase 3 trial, results indicated use of sotatercept was associated with a greater change from baseline in 6-minute walking distance from baseline to week 24 relative to placebo therapy.
Related Coverage: FDA Approves Sotatercept (Winrevair) for Pulmonary Arterial Hypertension

9. Vadadustat (Vafseo)

Date: March 27, 2024
Indication: Anemia Due to Chronic Kidney Disease
Background: Approval of vadadustat was awarded to Akebia Therapeutics based on data from the INNO2VATE program and an assessment of post-marketing safety data from Japan where vadadustat was launched in August 2020.
Related Coverage: FDA Approves Vadadustat (Vafseo) Tablets for Anemia Due to CKD in Adult Patients on Dialysis

10. Danicopan (Voydeya)

Date: March 29, 2024
Indication: Extravascular Hemolysis with Paroxysmal Nocturnal Hemoglobinuria (PNH)
Background: Approval of danicopan was awarded to AstraZeneca based on the ALPHA trial. Results of the indicated treatment with danicopan met the trial’s primary endpoint of increased hemoglobin levels from baseline to week 12 relative to placebo therapy.
Related Coverage: FDA Approves Danicopan as Add-On Therapy for Extravascular Hemolysis in PNH

11. Ceftobiprole Medocaril Sodium (Zevtera)

Date: April 03, 2024
Indication: Certain Bloodstream Infections, Bacterial Skin and Associated Tissue infections, and community-acquired bacterial pneumonia
Background: Approval of ceftobiprole medocaril sodium was awarded to Basilea Pharmaceutica International based on the ERADICATE trial. A 390-patient phase 3 trial, results concluded ceftobiprole was noninferior to daptomycin for overall treatment success in patients with complicated S. aureus bacteremia.
Related Coverage: Ceftobiprole Receives FDA Approval, Enhancing Antibiotic Options

12. Pegulicianine (Lumisight)

Date: April 17, 2024
Indication: Optical Imaging Agent for the Detection of Cancerous Tissue
Background: Approval of pegulicianine was awarded to Lumicell based on the INSITE trial. Results of the trial, which assessed margin status with or without pegulicianine fluorescence-guided surgery (pFGS), concluded use of pFGS in breast cancer surgery met prespecified thresholds for removal of residual tumor and specificity but did not meet the prespecified threshold for sensitivity.
Related Coverage: FDA Approves Pegulicianine-Based Imaging for Breast Cancer Surgery

13. Nogapendekin Alfa Inbakicept-pmln (Anktiva)

Date: April 22, 2024
Indication: Bladder Cancer
Background: Approval of Nogapendekin alfa inbakicept-pmln was awarded to ImmunityBio based on the QUILT-3.032 trial. An analysis of the open-label, phase 2/3 trial suggested complete responses were achieved with a persistence of effect, cystectomy avoidance, and 100% bladder cancer–specific survival at 24 months.
Related Coverage: FDA Approves Nogapendekin Alfa Inbakicept Plus BCG for BCG-Unresponsive NMIBC

14. Tovorafenib (Ojmeda)

Date: April 23, 2024
Indication: Relapsed or Refractory Pediatric Low-Grade Glioma
Background: Approval of tovorafenib was awarded to Day One Bio based on data from the ongoing FIREFLY-1 trial. Tovorafenib is the first agent indicated for patients with pediatric low-grade glioma.
Related Coverage: Sarah Leary, MD, MS: FDA Approval of Tovorafenib for Pediatric R/R BRAF+ Low-Grade Glioma (Preview Below)

15. Mavorixafor (Xolremdi)

Date: April 26, 2024
Indication: Warts, Hypogammaglobulinemia, Infections and Myelokathexis (WHIM) Syndrome
Background: Approval of mavorixafor was awarded to X4 Pharmaceuticals and supported by data from the 4WHIM trial. A 31-patient trial, results indicated improvement in absolute neutrophil counts, improvement in absolute lymphocyte counts, and a reduction in infections at week 52 relative to placebo. The approval marked the first indication for WHIM syndrome in FDA history.
Related Coverage: FDA Approves Mavorixafor as First Drug for WHIM Syndrome

16. Tarlatamab-dlle (Imdelltra)

Date: May 16, 2024
Indication: Extensive Stage Small Cell Lung Cancer
Background: Approval of tarlatamab was awarded to Amgen based on the DeLLphi-301 trial. Results of the phase 2, open-label, multicenter, multi-cohort trial suggested use of tarlatamab was associated with improved antitumor activity with durable objective responses and favorable survival outcomes in patients with previously treated small-cell lung cancer.
Related Coverage: Taofeek Owonikoko, MD, PhD, on the Impact of the FDA Approval of Tarlatamab in ES-SCLC (Preview Below)

17. Imetelstat (Rytelo)

Date: June 06, 2024
Indication: Low- to Intermediate-1 Risk Myelodysplastic Syndromes
Background: Approval of imetelstat was awarded to Geron Corporation based on the IMerge trial. A 178-patient phase 3 trial, results indicated use of imetelstat was associated with a greater red blood cell transfusion independence rate among patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents.
Related Coverage: Michael Sekeres, MD, on the FDA Approval of Imetelstat in Lower-Risk MDS With Anemia (Preview Below)

18. Elafibranor (Iqirvo)

Date: June 10, 2024
Indication: Primary Biliary Cholangitis (PBC)
Background: Approval of elafibranor was awarded to Ipsen based on the ELATIVE trial. A 161-patient double-blind, placebo-controlled, phase 3 trial, results demonstrated use of elafibranor was associated with significantly greater improvements in relevant biochemical indicators of cholestasis than placebo therapy at 52 weeks.
Related Coverage: HCPLive Special Report: Updates and Unmet Needs in PBC (Preview Below)

19. Sofpironium (Sofdra)

Date: June 18, 2024
Indication: Primary Axillary Hyperhidrosis
Background: Approval of sofpironium topical gel 12.45% was awarded to Botanix Pharmaceuticals based on the CARDIGAN studies. A pair of phase 3 studies, both trials met all primary and secondary endpoints, with clinically and statistically meaningful changes from baseline in Gravimetric Sweat Production (GSP) and the Hyperhidrosis Disease Severity Measure-Axillary, 7-item (HDSM-AX7) score.
Related Coverage: FDA Approves Sofpironium, First Treatment for Primary axillary Hyperhidrosis

20. Crovalimab-akkz (Piasky)

Date: June 20, 2024
Indication: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Background: Approval of crovalimab was awarded to Genentech based on the COMMODORE 2 trial. A 204-patient, randomized, open-label, phase 3 trial, results indicated crovalimab was noninferior to eculizumab for both hemolysis control and transfusion avoidance.
Related Coverage: FDA Approves Crovalimab for Paroxysmal Nocturnal Hemoglobinuria

References:

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