News|Articles|July 1, 2026
FDA News Recap: Novel Drug Approvals in First Half of 2026
Author(s)Abigail Brooks, MA
Fact checked by: Victoria Johnson
Key Takeaways
- Copper histidinate, pegzilarginase, navepegritide, and tividenofusp alfa advanced rare-disease care using open-label and randomized datasets, with approvals frequently anchored to biochemical normalization and surrogate outcomes.
- Oncology additions included vepdegestrant (ESR1-mutated ER+/HER2– disease), relacorilant plus nab-paclitaxel (platinum-resistant ovarian cancer), sonrotoclax (post-BTKi MCL), and pivekimab sunirine for BPDCN.
A review of 22 novel drugs approved by the US Food and Drug Administration during the first half of 2026, with links to coverage from MJH Life Sciences publications.
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The first half of 2026 brought a wave of innovation across the medical community, with 22 novel drug approvals signaling major strides in disease treatment and management across a wide range of specialties.
From new options in oncology, including therapies for breast cancer, ovarian cancer, and mantle cell lymphoma, to groundbreaking advances in rare and ultra-rare diseases like Menkes disease, Hunter syndrome, and Arginase 1 Deficiency, this wave of approvals underscores the growing precision and diversity of today's therapeutic landscape. Several approvals also opened doors in historically underserved areas, including chronic hepatitis delta virus infection, thyroid eye disease, and once-weekly basal insulin dosing. Together, these developments signal not just short-term progress, but a promising trajectory for the rest of 2026 and for the future of patient care across specialties.
To celebrate the first half of 2026, we have compiled a recap for each of the 22 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, Contemporary Pediatrics, Contemporary OB/GYN, and others.
1. Copper Histidinate (Zycubo)
Date: January 12, 2026
Indication: To treat Menkes disease
Background: Approval supported by data from 2 completed open-label, single-arm, single-site studies in which 129 patients with Menkes disease were treated with copper histidinate, formerly known as CUTX-101. In the studies, treatment consisted of subcutaneous administration of copper histidinate (1450 μg, equivalent to 250 μg elemental copper) twice daily until aged 12 months and once daily thereafter, for a total treatment duration of up to 3 years
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2. Difamilast (Adquey)
Date: February 12, 2026
Indication: To treat mild to moderate atopic dermatitis
Background: Approval supported by phase 3 clinical trial data in adult and pediatric patients with mild to moderate AD. Patients applying difamilast ointment twice daily achieved statistically significant improvements in Investigator’s Global Assessment (IGA) scores—the primary end point—vs vehicle at week 4, with a meaningful proportion achieving IGA 0 (clear) or 1 (almost clear) with at least a 2-grade improvement from baseline. Secondary endpoints, including Eczema Area and Severity Index scores and patient-reported pruritus, also favored difamilast, with itch reductions observed early in treatment.
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3. Milsaperidone (Bysanti)
Date: February 20, 2026
Indication: To treat schizophrenia and to treat manic or mixed episodes associated with bipolar I disorder
Background: Approval supported by bioequivalence data and the existing clinical database for iloperidone, which was already approved for schizophrenia and manic or mixed episodes associated with adult bipolar I disorder.
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4. Pegzilarginase-nbln (Loargys)
Date: February 23, 2026
Indication: To treat hyperarginemia in adults and pediatric patients two years and older with Arginase 1 Deficiency, in conjunction with dietary protein restriction
Background: Approval supported by data from the multicenter, randomized, double-blind, placebo-controlled phase 3 PEACE trial (NCT03921541). At 24 weeks, pegzilarginase produced a significant reduction in plasma arginine levels compared with placebo, lowering geometric mean levels from 354.0 to 86.4 μmol/L and normalizing plasma arginine in 90.5% of treated patients, whereas no normalization was observed in the placebo group.
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5. Navepegritide (Yuviwel)
Date: February 27, 2026
Indication: To increase linear growth in pediatric patients 2 years and older with achondroplasia with open epiphyses
Background: Approval supported by data from 3 randomized, double-blind, placebo-controlled trials of navepegritide, including the pivotal phase 3 ApproaCH study, as well as open-label extension data of up to 3 years. Increased AGV was used as a surrogate outcome reasonably likely to predict clinical benefit.
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6. Linerixibat (Lynavoy)
Date: March 17, 2026
Indication: To treat cholestatic pruritus associated with primary biliary cholangitis
Background: Approval based on data from the global phase 3 GLISTEN trial, which met both primary and key secondary endpoints, demonstrating significant, rapid and sustained improvements in cholestatic pruritus and itch-related sleep interference versus placebo.
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7. Icotrokinra (Icotyde)
Date: March 17, 2026
Indication: To treat moderate-to-severe plaque psoriasis in patients 12 years and older who weigh at least 40 kg and who are candidates for systemic therapy or phototherapy
Background: Approval based on results from 4 phase 3 trials in the ICONIC program, enrolling approximately 2500 patients aged 12 and older. The program began with ICONIC-LEAD, a multicenter, randomized, double-blind, placebo-controlled study that first established the agent's efficacy and safety profile across adults and adolescents.
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8. Tividenofusp alfa-eknm (Avlayah)
Date: March 24, 2026
Indication: To treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II)
Background: Approval supported by data from an ongoing phase 1/2, open-label study of 47 patients with Hunter syndrome. At 24 weeks, treatment led to geometric mean reductions of –91.4% in cerebrospinal fluid (CSF) heparan sulfate and –87.9% in urinary heparan sulfate, with normalization achieved in most patients. These biomarker changes were accompanied by reductions in serum neurofilament light (NfL) chain, including –21.2% at week 49 and –70.5% at week 104, suggesting a potential effect on neuroaxonal injury.
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9. Relacorilant (Lifyorli)
Date: March 25, 2026
Indication: To treat platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after one to three prior systemic treatment regimens, at least one of which included bevacizumab
Background: Approval supported by data from the phase 3 ROSELLA trial (NCT05257408), which enrolled patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who had received a maximum of 3 prior lines of systemic therapy, including prior bevacizumab (n = 381). In this trial, patients who received relacorilant plus nab-paclitaxel achieved a median progression-free survival (PFS) of 6.5 months (95% CI, 5.6-7.4) vs 5.5 months (95% CI, 3.9-5.9) among those who received nab-paclitaxel alone (HR, 0.70; 95% CI, 0.54-0.91; 2-sided P = .0076).
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10. Insulin icodec-abae (Awiqli)
Date: March 26, 2026
Indication: To improve glycemic control in adults with type 2 diabetes mellitus
Background: Approval based on results from the ONWARDS type 2 diabetes phase 3a program, which comprised 5 randomized, active-controlled, treat-to-target clinical trials in approximately 4000 adults with T2D evaluating insulin icodec head-to-head against standard once-daily basal insulins, including insulin glargine U100 and insulin degludec.
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11. Orforglipron (Foundayo)
Date: April 1, 2026
Indication: To reduce excess body weight and maintain weight reduction long term in adults with obesity or adults with overweight in the presence of at least one weight-related comorbid condition, in combination with a reduced-calorie diet and increased physical activity
Background: Approval based on findings from the ATTAIN Phase 3 global clinical development program. In ATTAIN-1, individuals taking the highest dose of orforglipron and who stayed on treatment lost an average of 27.3 pounds (12.4%) compared to 2.2 pounds (0.9%) with placebo. Participants taking orforglipron, regardless of trial completion, lost an average of 25 pounds (11.1%), compared to 5.3 pounds (2.1%) with placebo. Positive topline results from ATTAIN-2 showed all 3 doses of orforglipron met the trial’s primary and all key secondary endpoints, delivering significant weight loss, meaningful A1C reductions, and improvements in cardiometabolic risk factors at 72 weeks in adult patients with with obesity or overweight and type 2 diabetes.
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12. Doravirine and islatravir (Idvynso)
Date: April 20, 2026
Indication: To treat HIV-1 infection (as a complete regimen) in adults to replace the current antiretroviral regimen in those who are virologically-suppressed on a stable antiretroviral regimen with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine
Background: Approval supported by findings from 2 phase 3 trials involving more than 700 adults with virologic suppression. Across both studies, the regimen met its primary end point, showing noninferior efficacy compared with both bictegravir/emtricitabine/tenofovir alafenamide and baseline antiretroviral therapies at 48 weeks.
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13. Vepdegestrant (Veppanu)
Date: May 1, 2026
Indication: To treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy
Background: Approval backed by data from the phase 3 VERITAC-2 trial (NCT05654623), which demonstrated that patients harboring ESR1 mutations treated with vepdegestrant (n = 136) experienced a median progression-free survival (PFS) of 5.0 months (95% CI, 3.7-7.4) compared with 2.1 months (95% CI, 1.9-3.5) for those treated with fulvestrant (Faslodex; n = 134; HR 0.57; 95% CI, 0.42-0.77; P = 0.0001).1,3 The overall response rates (ORRs) in these respective groups were 19% (95% CI, 12%-27%) and 4% (95% CI, 1.6%-10%).
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14. Sonrotoclax (Beqalzi)
Date: May 13, 2026
Indication: To treat adults with relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase inhibitor
Background: Approval supported by data from the phase 1/2 BGB-11417-201 trial (NCT05471843), which showed that patients with relapsed/refractory MCL who were previously treated with an anti-CD20–based therapy and a BTK inhibitor (n = 103) experienced an overall response rate (ORR) of 52% (95% CI, 42%-62%) and a median time to response of 1.9 months. At a median follow-up of 11.9 months, the median duration of response was 15.8 months (95% CI, 7.4-not estimable).
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15. Baxdrostat (Baxfendy)
Date: May 15, 2026
Indication: To treat hypertension in combination with other antihypertensive drugs
Background: Approval based on results from the phase 3 BaxHTN trial, which showed mean seated systolic blood pressure fell by 15.7 mm Hg in the 2-mg group and 14.5 mm Hg in the 1-mg group, compared with 5.8 mm Hg in the placebo group. Placebo-adjusted reductions were 9.8 mm Hg (95% CI, -12.6 to -7.0; P <.001) for 2 mg and 8.7 mm Hg (95% CI, -11.5 to -5.8; P <.001) for 1 mg.
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16. Bulevirtide-gmod (Hepcludex)
Date: May 22, 2026
Indication: To treat chronic hepatitis delta virus infection in adults without cirrhosis or with compensated cirrhosis
Background: Approval supported by data from MYR301, a multicenter, randomized, open-label, parallel-arm phase 3 trial. At week 48, combined response was achieved in 48% of patients in the bulevirtide group compared with 2% in the delayed-treatment group.¹ The 46-percentage-point difference formed the basis for the accelerated approval, with combined response serving as the primary efficacy endpoint.
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17. Pivekimab sunirine-pvzy (Decnupaz)
Date: May 27, 2026
Indication: To treat adults with blastic plasmacytoid dendritic cell neoplasm
Background: Approval backed by data from the phase 1/2 CADENZA trial (NCT03386513), which showed that at a median follow-up of 21.5 months, treatment-naive patients with BPDCN (n = 33) experienced a complete remission/clinical complete remission (CR/CRc) rate of 69.7% (95% CI, 51.3%-84.4%) and a median duration of CR/CRc of 9.7 months (95% CI, 2.9-not estimable [NE]).
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18. Cefepime and Zidebactam (Zaynich)
Date: May 29, 2026
Indication: To treat complicated urinary tract infections, including pyelonephritis, caused by designated susceptible microorganisms
Background: Approval based in part on findings from the phase 3 ENHANCE-1 trial, in which the intravenous antibiotic achieved a higher composite clinical cure and microbiologic response rate than meropenem at the test-of-cure visit.
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19. Ensitrelvir (Xocova)
Date: May 29, 2026
Indication: To use as post-exposure prophylaxis of coronavirus disease 2019 (COVID-19) following contact with an individual who has COVID-19
Background: Approval based on results from SCORPIO-PEP, a global phase 3, randomized, double-blind, placebo-controlled study evaluating ensitrelvir for postexposure prevention of COVID-19. Results showed that ensitrelvir reduced the risk of symptomatic COVID-19 by 67% through day 10 compared with placebo. The treatment was generally well tolerated, with adverse event rates similar between the ensitrelvir and placebo groups.
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20. Gadoquatrane (Ambelvist)
Date: June 12, 2026
Indication: To detect and visualize lesions with abnormal vascularity, in conjunction with MRI
Background: Approval based on data from the global pivotal phase 3 QUANTI program. In QUANTI CNS, gadoquatrane met the primary endpoints of non-inferiority to comparator mGBCAs (gadobutrol, gadoterate meglumine, gadoteridol) across 3 established visualization parameters: contrast enhancement, delineation, and morphology, all assessed by blinded independent readers. Gadoquatrane also demonstrated non-inferior sensitivity and specificity for lesion detection and exclusion, and showed superiority over unenhanced MRI across the same visualization endpoints.
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21. Tebipenem pivoxil (Utebzi)
Date: June 17, 2026
Indication: To treat complicated urinary tract infections, including pyelonephritis, caused by several susceptible microorganisms in adults who have limited or no alternative oral treatment options
Background: Approval based on results from the phase 3 PIVOT-PO trial (NCT06059846), which met its primary end point by showing that tebipenem pivoxil was noninferior to imipenem-cilastatin in regard to the overall response rate (composite of clinical cure plus microbiological eradication) at the test-of-cure visit in hospitalized adult patients with cUTI.
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22. Veligrotug-vvze (Lumvoa)
Date: June 26, 2026
Indication: To treat thyroid eye disease
Background: Approval based on positive results from the THRIVE and THRIVE-2 clinical trials investigating the intravenous insulin-like growth factor 1 receptor (IGF-1R) antagonist administered as 5 infusions every 3 weeks over 12 weeks. Both studies met their primary endpoints and all secondary endpoints, with statistically significant improvements at week 15 across key signs and symptoms of TED.
Related Coverage:
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