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Roundup: Novel Drug Approvals in 2024
In the first half of 2024, the FDA approved 23 novel drugs for conditions including alopecia areata, Alzheimer disease, small cell lung cancer, bladder cancer, and more.
In the first half of 2024, the FDA has approved 23 novel drugs, including indications for alopecia areata, Alzheimer disease, small cell lung cancer, bladder cancer, and more. In the first 6 months alone, there were 20 novel approvals.
Here is a recap of all novel drug approvals from the first half of 2024, including related coverage of each drug.
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1. Berdazimer (Zelsuvmi; Ligand)
Date: January 5, 2024
Indication: The treatment of molluscum contagiosum in individuals aged 1 year and older.
Background: In 2 phase 3 trials, B-SIMPLE 4 and B-SIMPLE 2, the results showed the drug was well tolerated when used once a day. The trials were included in the B-SIMPLE phase 3 program, which enrolled 1598 individuals.
Related Coverage: FDA Approves Berdazimer Topical Gel as First Treatment for Molluscum Contagiosum
2. Cefepime, Enmetazobactam (Exblifep; Allecra)
Date: February 22, 2024
Indication: To treat complicated urinary tract infections.
Background: Supported by data from the phase 3 ALLIUM trial, which demonstrated the non-inferiority and superiority of cefepime/enmetazobactam vs piperacillin/tazobactam in regard to the primary composite outcome measure of clinical cure and microbiological eradication in patients with cUTIs.
Related Coverage: FDA approves cefepime/enmetazobactam for complicated UTI
3. LetibotulinumtoxinA-wlbg (Letybo; Hugel America)
Date: February 29, 2024
Indication:To temporarily improve the appearance of moderate-to-severe glabellar lines.
Background: The approval comes after the FDA initially sent a Complete Response Letter last year in response to the company Biologics License Application filed in October 2022. Hugel then sent out an additional BLA at the end of August, reviewing management of its facility and supplementary work in-line from requests received from the FDA in its CRL.
Related Coverage: FDA Approves Hugel America’s Letybo to Treat Moderate-to-Severe Frown Lines in Adults
4. Tislelizumab-jsgr (Tevimbra; BeiGene)
Date: March 13, 2024
Indication: Monotherapy for the treatment of adult patients with unresectable or metastatic esophageal squamous cell carcinoma after prior systemic chemotherapy.
Background: In the phase 3, global, randomized, open-label, RATIONALE 302 trial, investigators assessed the efficacy and safety of tislelizumab-jsgr when compared with investigator’s choice of chemotherapy as a second-line treatment for patients with unresectable, locally advanced or metastatic ESCC. The median overall survival (OS) in the ITT population in the tislelizumab-jsgr arm was 8.6 months compared to 6.3 months in the chemotherapy arm.
Related Coverage: FDA Approves Tislelizumab-jsgr for Advanced or Metastatic Esophageal Squamous Cell Carcinoma After Prior Chemotherapy
5. Resmetirom (Rezdiffra; Madrigal Pharmaceuticals)
Date: March 14, 2024
Indication: Treatment of adults with nonalcoholic steatohepatitis with moderate to advanced liver fibrosis, in conjunction with diet and exercise.
Background: Based on results from the phase 3, ongoing pivotal, multicenter, randomized, double-blind, placebo-controlled MAESTRO-NASH trial. Following 52 weeks of treatment on doses of 100 mg and 80 mg, resmetirom showed statistically significant improvement compared to placebo on 2 primary endpoints: NASH resolution with no worsening of fibrosis, and an improvement in fibrosis by at least one stage with no worsening of the NAFLD activity score.
Related Coverage: FDA Grants Accelerated Approval of Resmetirom for Noncirrhotic Nonalcoholic Steatohepatitis With Moderate to Advanced Liver Fibrosis
6. Aprocitentan (Tryvio; Idorsia)
Date: March 19, 2024
Indication:Treatment of hypertension in combination with other antihypertensive drugs to lower blood pressure in adults whose blood pressure is not adequately controlled on other medications.
Background: The drug was approved as a monotherapy in a phase 3 study for patients with hypertension and as an add on therapy in the phase 3 PRECISION (NCT03541174) study for patients with confirmed resistant hypertension. Investigators found that the 12.5 mg dosage was superior to the placebo through week 4, with the effect being consistent for sitting diastolic blood pressure.
Related Coverage: FDA Approves Aprocitentan for Treatment of Hypertension in Combination With Other Drugs
7. Givinostat (Duvyzat; ITF Therapeutics)
Date: March 21, 2024
Indication: Treatment of Duchenne muscular dystrophy (DMD) in patients aged 6 years and older.
Background: The approval was based on the multicenter, randomized, double-blinded phase 3 EPIDYS trial. After 18 months of treatment, those taking givinostat demonstrated statistically significant less decline in the time it took to climb 4 stairs compared to the placebo.
8. Sotatercept-csrk (Winrevair; Merck)
Date: March 26, 2024
Indication: Treatment of adults with pulmonary arterial hypertension.
Background: The approval is based on the STELLAR phase 3 trial comparing sotatercept-csrk with the placebo in combination with background standard of care therapies in adults with PAH.
Related Coverage: FDA Approves Sotatercept-csrk for Pulmonary Arterial Hypertension
9. Vadadustat (Vafseo; Akebia Therapeutics)
Date: March 27, 2024
Indication: The treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least 3 months.
Background: The approval of vadadustat is based on data from the INNO2VATE program and assessment of post-marketing safety data from Japan, where the drug launched in August 2020.
Related Coverage: FDA Approves Vadadustat for Oral Treatment of Anemia Due to Chronic Kidney Disease
10. Danicopan (Voydeya; AstraZeneca)
Date: March 29, 2024
Indication: The treatment of extravascular hemolysis in adult patients with paroxysmal nocturnal hemoglobinuria.
Background: The double-blind ALPHA trial evaluated danicopan. According to the findings, patients who received danicopan in addition to their C5 inhibitor therapy showed better changes in hemoglobin from baseline to week 12. The investigators noted that the first sign of improvements was observed by week 2 and was maintained through week 12.
Related Coverage: FDA Approves Danicopan With C5 Inhibitor Therapy to Treat EVH in Adults With Paroxysmal Nocturnal Hemoglobinuria
11. Ceftobiprole Medocaril Sodium (Zevtera; Basilea Pharmaceutical)
Date: April 3, 2024
Indication: To treat certain bloodstream infections, bacterial skin and associated tissue infections, and community-acquired bacterial pneumonia.
Background: The approval was based on 3 randomized clinical trials investigating the drug for each of the indications. The trials showed positive results, with each meeting the primary end point of the respective trial.
Related Coverage: FDA Approves Zevtera as Antibiotic to Treat 3 New Indications
12. Lumisight (pegulicianine)
Date: April 17, 2024
Indication: To use as an optical imaging agent for the detection of cancerous tissue.
Background: The INSITE trial showed that Lumisight had an 84% diagnostic accuracy in detecting residual cancer in real-time, including disease that may have been missed during lumpectomy. The study enrolled patients at least 18 years of age or older who were undergoing lumpectomy for stage I to III invasive breast cancer and/or ductal carcinoma in situ.
Related Coverage: FDA Approves Lumisight and Lumicell DVS for Residual Breast Cancer Detection
13. Nogapendekin alfa inbakicept-pmln (Anktiva, N-803; ImmunityBio Inc)
Date: April 22, 2024
Indication: To treat bladder cancer.
Background: The approval was based on the QUILT-3.032 study, which was a single-arm, multicenter trial that included 77 adults with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma with or without papillary tumors after a transurethral resection. Study results showed there were approximately 62% of individuals who achieved a complete response, with 58% having a duration of over 12 months and 40% having a duration of over 24 months.
Related Coverage: FDA Approves Anktiva Plus Bacillus Calmette-Guérin For Non-Muscle Invasive Bladder Cancer
14. Tovorafenib (Ojemda; Day One Biopharmaceuticals)
Date: April 23, 2024
Indication: To treat relapsed or refractory pediatric low-grade glioma.
Background: The approval was based on data from the phase 2 FIREFLY-1 trial, showing patients experienced an overall response rate of 51%, with a median duration of response of 13.8 months. The approval marks the first systemic therapy for pediatric patients with low-grade glioma with BRAF rearrangements.
Related Coverage: FDA Grants Accelerated Approval to Tovorafenib for Pediatric Relapsed/Refractory BRAF+ Low-Grade Glioma
15. Mavorixafor (Xolremdi; X4 Pharmaceuticals)
Date: April 26, 2024
Indication: To treat WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis).
Background: The approval was based on the 4WHIM study, with the study evaluating the safety and efficacy of the drug for patients aged 12 and older. The drug group experienced an approximate 40% reduction in the total infection score and 60% lower infections rates when compared with the placebo.
Related Coverage: FDA Approves Mavorixafor Capsules to Treat Patients With WHIM Syndrome
16. Tarlatamab-dlle (Imdelltra; Amgen)
Date: May 16, 2024
Indication: To treat extensive stage small cell lung cancer.
Background: The approval was based on results from the DELLphi-301 trial. The results showed that the primary end points were met, showing an overall response rate was 40% and the median duration of response was 9.7 months. Disease control, progression-free survival, overall survival, safety, and serum concentration levels were also evaluated in the trial.
Related Coverage: FDA Grants Accelerated Approval to Tarlatamab for ES-SCLC
17. Imetelstat (Rytelo; Geron Corp)
Date: June 16, 2024
Indication: To treat low- to intermediate-1 risk myelodysplastic syndromes.
Background: The approval was based on results from the IMerge phase 3 clinical trial, meeting the primary and key secondary end points. Further, the drug showed significantly higher rates of red blood cell-transfusion independence compared with the placebo for at least 8 consecutive weeks for at least 24 weeks.
Related Coverage: FDA Approves Imetelstat for Lower-Risk Myelodysplastic Syndromes With Transfusion-Dependent Anemia
18. Elafibranor (Iqirvo; GENFIT, Ipsen)
Date: June 10, 2024
Indication: To treat primary biliary cholangitis in combination with ursodeoxycholic acid.
Background: Previous results of a study showed that elafibranor decreases the toxic effects of bile acid and inflammation by targeting PPAR-α and PPAR-δ. The approval was based on a study of 161 individuals, which showed that a biochemical response was observed in 51% of patients on elafibranor compared with the 4% of the placebo.
Related Coverage: FDA Grants Accelerated Approval for Elafibranor as Treatment of Primary Biliary Cholangitis
19. Sofpironium (Sofdra; Botanix)
Date: June 18, 2024
Indication: To treat primary axillary hyperhidrosis.
Background: The approval was based on 2 phase 3 trials in the CARDIGAN program that evaluated safety and efficacy of sofpironium. The primary outcomes of both studies were the number of individuals who experienced a 2 point or greater improvement in Hyperhidrosis Disease Severity Measure-Axillary 7 Item total score from baseline to the end of treatment, meeting all primary and secondary endpoints with statistically meaningful changes in the score.
Related Coverage: FDA Approves Sofpironium, First Treatment for Primary Axillary Hyperhidrosis
20. Crovalimab-akkz (Piasky; Genentech)
Date: June 20, 2024
Indication: To treat paroxysmal nocturnal hemoglobinuria.
Background: The approval was based on the COMMODORE 2 trial, including patients with documented PNH per high-sensitivity flow cytometry with a body weight of at least 40 kg at the time of screening. The results showed that the rate of hemolysis control form week 5 to week 25 achieved approximately 79.3% patients treated with crovalimab compared with 79% for those receiving eculizumab.
Related Coverage: FDA Approves Crovalimab for Paroxysmal Nocturnal Hemoglobinuria
21. Ensifentrine (Ohtuvayre; Verona Pharma)
Date: June 26, 2024
Indication: To treat chronic obstructive pulmonary disease.
Background: The results of the ENHANCE trials contributed to the approval, finding that ensifentrine improved chronic obstructive pulmonary disease symptoms and quality of life. Further, there was a lack of adverse effects for the treatment, showing the drog was well tolerated and was not associated with gastrointestinal issues or an increase in pneumonia.
Related Coverage: FDA Approves Ensifentrine for Maintenance Treatment of Chronic Obtrusive Pulmonary Disorder
22. Donanemab-azbt (Kisunla; Eli Lilly and Company)
Date: July 2, 2024
Indication: To treat Alzheimer’s disease.
Background: The approval was based on the TRAILBLAZER-ALZ 2 showed donanemab significantly slowed cognitive progression at 76 weeks, with 23 of 24 outcomes having statistical significance. The incidence of serious adverse events was 17.4% for those receiving donanemab and 25.8% for the placebo.
Related Coverage: FDA Approves Donanemab-Azbt for Early Symptomatic Alzheimer Disease
23. Deuruxolitinib (Leqselvi; Sun Pharmaceutical Industries Ltd)
Date: July 25, 2024
Indication: To treat severe alopecia areata.
Background: The FDA’s approval was based on the THRIVE-AA1 and the THRIVE-AA2 trials, which included individuals with alopecia areata who had at least 50% of scalp hair loss. The primary end point of Severity of Alopecia Tool score of 20 less was met, with 30% of individuals experiencing 80% or more scalp hair coverage.
Related Coverage: FDA Approves Deuruxolitinib for the Treatment of Severe Alopecia Areata
