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FDA Decisions Expected: April 2026

Your monthly synopsis of new drugs expected to hit the market 

March 15, 2026

Drug pipeline for April 2026

At Prime Therapeutics (Prime), we have positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs awaiting approval by the United States (U.S.) Food and Drug Administration (FDA).
2Q 2026: baxdrostat 

The FDA granted a Priority Review for AstraZeneca’s baxdrostat, an oral once daily aldosterone synthase inhibitor for the treatment of uncontrolled or resistant hypertension (uHTN or rHTN), defined as hypertension that persists despite treatment with two (uHTN) or three (rHTN) antihypertensive medications. In the BaxHTN trial, the addition of baxdrostat 1 mg or 2 mg to standard therapy significantly reduced seated systolic blood pressure (SBP) at 12 weeks versus placebo (placebo-adjusted difference, -8.7 mmHg and -9.8 mmHg, respectively; p<0.0001) and increased the proportion of patients achieving controlled seated SBP (< 130 mmHg) compared to placebo (39.4% and 40% versus 18.7%, respectively; p<0.0001).¹ In the Bax24 trial, baxdrostat 2 mg lowered 24-hour ambulatory systolic blood pressure (ASBP) by 14 mmHg compared to placebo at week 12 (p<0.0001).² If approved, baxdrostat will be the first aldosterone synthase inhibitor indicated for uHTN or rHTN. 

For more information, see the baxdrostat Deep Dive in the January 2026 edition of Prime’s Quarterly Drug Pipeline. 

2Q 2026: pegargiminase (ADI-PEG 20)
DB-OTO is an investigational cell-selective, dual adeno-associated virus (AAV) vector gene therapy for the treatment of congenital hearing loss caused by variants in the otoferlin (OTOF) gene. The FDA granted DB-OTO Fast Track, Orphan Drug, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations as well as a Commissioner’s National Priority Voucher which may shorten the FDA review timeline to about one to two months. The open-label, single-arm, Phase 1/2 CHORD trial demonstrated that nine out of 12 children with profound hearing loss due to OTOF gene variants who were treated with a single DB-OTO intracochlear infusion experienced hearing improvements at a threshold of ≤ 70 dB HL (primary endpoint) as assessed by behavioral pure tone audiometry (PTA).³ If approved, DB-OTO will be the first pharmacologic treatment for congenital hearing loss due to variants of the OTOF gene and may provide an alternative treatment to cochlear implants in patients with the condition.
 
For more information, see the DB-OTO Deep Dive in the January 2026 edition of Prime’s Quarterly Drug Pipeline.
1H 2026: tebipenem HBr
Polaris is awaiting an FDA decision for pegargiminase, an arginine degrading enzyme, for the treatment of malignant pleural mesothelioma (MPM) with non-epithelioid histology. The FDA granted the drug Fast Track and Orphan Drug designations. The Phase 2/3 ATOMIC-Meso trial randomized patients with MPM to weekly intramuscular (IM) pegargiminase or placebo, each in combination with pemetrexed and platinum-containing chemotherapy as first-line treatment. The study reported a median overall survival (OS) of 9.3 months in the pegargiminase group compared with 7.7 months in the placebo group (hazard ratio [HR], 0.71; p=0.02).³ The median progression-free survival (PFS) was 6.2 months with pegargiminase compared with 5.6 months with placebo (HR, 0.65; p=0.02). If approved, pegargiminase may be an effective addition to standard platinum and pemetrexed chemotherapy in patients with MPM.
2Q 2026: pivekimab sunirine (PVEK)
Abbvie has submitted a New Drug Application (NDA) for PVEK, a first-in class CD123-targeting antibody-drug conjugate, for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). The FDA granted it a Breakthrough Therapy designation. The NDA was supported by interim data from the open-label, Phase 1/2 CADENZA trial that showed among untreated patients, PVEK monotherapy led to a composite complete response (CR) rate (defined as CR + clinical CR with minimal skin abnormality) of 70%, a median duration of composite CR of 9.8 months, an overall response rate (ORR) of 85% and a median OS of 16.6 months.⁴ In patients with relapsed/refractory (R/R) BPDCN, the composite CR rate was 14%, the median duration of composite CR was 9.2 months, the ORR was 35% and median OS was 5.8 months. PVEK was administered in an outpatient setting as an intravenous (IV) infusion over less than 30 minutes on day 1 of a 21-day cycle. PVEK was associated with a manageable safety profile. If approved, PVEK would be the second CD123-targeting therapy for BPDCN following Stemline’s tagraxofusp-erzs (Elzonris).
2Q 2026: sonrotoclax
BeOne is awaiting an FDA decision for sonrotoclax for the treatment of R/R mantle cell lymphoma (MCL) following treatment with a Bruton’s tyrosine kinase (BTK) inhibitor. Sonrotoclax is a next-generation B-cell lymphoma 2 (BCL2) inhibitor. The FDA has granted the agent Breakthrough Therapy, Fast Track and Orphan Drug designations and a Priority Review. In an ongoing single-arm, Phase 1/2 dose-escalation/expansion trial (NCT05471843), at a follow-up of 10.1 months, as reported by an internal review board (IRB), sonrotoclax 320 mg once daily monotherapy led to an ORR of 53.4%, with a CR rate of 14.6%.⁵ The median time to response was 1.9 months, median duration of response (DOR) was 15.8 months and median PFS was 6.5 months. If approved, sonrotoclax will be the first BCL2 inhibitor that is indicated in patients with R/R MCL post-BTK inhibitor therapy. 

For more information, see the sonrotoclax Deep Dive in the January 2026 edition of Prime’s Quarterly Drug Pipeline.
2Q 2026: zidebactam/cefepime
Wockhardt’s zidebactam/cefepime, a ß-lactam enhancer/ß-lactam combination antibiotic, is under FDAs Priority Review for the treatment of complicated urinary tract infections (cUTI), including pyelonephritis, with or without concurrent bacteremia caused by Gram-negative bacteria including multidrug-resistant (MDR). It also has Fast Track and Qualified Infectious Disease Product designations. A Phase 3 non-inferiority trial (NCT04979806) randomized hospitalized adults with cUTI or acute pyelonephritis (AP) to zidebactam/cefepime or meropenem, each administered IV every eight hours.⁶ Zidebactam/cefepime was superior to meropenem based on the primary composite endpoint of clinical and microbiology cure at the test-of-cure visit (day 17 ± 2 days) (89% versus 68.4%, respectively). The secondary endpoint of overall success rate at the end-of-treatment (day 7–10 ± 1 day) was similar between zidebactam/cefepime and meropenem (96.1% versus 97.1%, respectively). If approved, zidebactam/cefepime would offer a new antibiotic option for treating cUTI caused by MDR Gram-negative pathogens.
April 5, 2026: tividenofusp alfa (DNL310)

Tividenofusp alfa is a next-generation enzyme replacement therapy (ERT), by Denali Therapeutics, for the treatment of patients with mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The FDA granted it Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and a Priority Review. Hunter syndrome is a rare, genetic, lysosomal disorder that causes progressive multisystem and neurologic decline with onset during childhood. Interim data from an open-label, Phase 1/2 trial reported weekly IV infusions of tividenofusp alfa significantly reduced cerebrospinal fluid (CSF) heparan sulfate (HS) by 91% and urinary HS by 88%─key biomarkers of Hunter syndrome that could support an Accelerated Approval. ⁷˒⁸˒⁹ A significant reduction in HS levels was seen at week 24 and maintained through week 201. Other secondary endpoints showed stabilization or improvement in adaptive behavior, cognition, hearing, and liver volume through week 201. If approved, tividenofusp alfa will be an alternative treatment to Takeda’s idursulfase (Elaprase). Unlike Elaprase, tividenofusp alfa crosses the blood-brain barrier but whether this results in improved central nervous system symptoms has not been studied in a clinical trial.

For more information, see the tividenofusp alfa Deep Dive in the October 2025 edition of Prime’s Quarterly Drug Pipeline.

April 6, 2026: allogeneic T-cell immunotherapy (Orca-T)

Orco Bio is awaiting an FDA decision for their allogeneic T cell immunotherapy, Orca-T, for use in patients with hematological malignancies. Orca-T contains purified regulatory T cells, hematopoietic stem cells and conventional T cells from matched related or unrelated donors. Orca-T was granted a Priority Review and a Regenerative Medicine Advanced Therapy designation by the FDA. In the Phase 3 Precision-T trial, Orca-T was administered IV after myeloablative conditioning followed by tacrolimus and significantly improved the rate of 12-month moderate to -severe chronic GVHD -free survival (primary endpoint) compared with standard allogeneic hematopoietic stem cell transplant (allo-HSCT) (78% versus 38.4%, respectively; p<0.00001).¹⁰ If approved, Orca-T may offer better outcomes compared to traditional allo-HSCT in adults with high-risk hematologic cancers. 

For more information, see the allogeneic T-cell immunotherapy (Orca-T) Deep Dive in the January 2026 edition of Prime’s Quarterly Drug Pipeline.

April 10, 2026: orforglipron

Orforglipron, Eli Lilly’s non-peptide glucagon-like peptide-1 (GLP-1) receptor agonist, is under FDA review for the treatment of obesity or overweight in adults. It was granted the FDA Commissioners’ National Priority Voucher (CNPV) which shortens the FDA time for review. Orforglipron is administered orally once daily. In the Phase 3 ATTAIN-1 trial, adults with obesity and without diabetes achieved significantly greater weight loss from baseline to week 72 with orforglipron 36 mg compared to placebo (11.2% versus 2.1%, respectively; p<0.001).¹¹ The most common adverse events reported with orforglipron were gastrointestinal in nature. If approved, orforglipron will be the second oral GLP-1 agent for weight loss and will compete with Novo Nordisk‘s oral semaglutide (Wegovy). 

For more information, see the orforglipron Deep Dive in the January 2026 edition of Prime’s Quarterly Drug Pipeline.

April 10, 2026: vusolimogene oderparepvec (RP1)

Replimune has resubmitted RP1, an engineered herpes simplex virus type 1 (HSV-1) oncolytic therapy, in combination with nivolumab (Opdivo) for the treatment of advanced melanoma that has progressed on anti-programmed cell death (PD)-1 treatment. RP1 is administered by intratumoral injection every two weeks for eight cycles, with Opdivo administered IV for 30 cycles. This is the second review for RP1 after the FDA issued a Complete Response Letter (CRL) in July 2025 stating that the open-label, Phase 2 IGNYTE trial did not provide substantial evidence of effectiveness. The resubmission included additional data and analyses. Data reported in November 2025 demonstrated an ORR of 33.6% and a median DOR of 24.8 months with the combination therapy. ¹² If approved, RP1 plus Opdivo would offer a new second-line option for advanced melanoma, an area with limited available treatments.

For more information, see the vusolimogene oderparepvec (RP1) Deep Dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline.

April 18, 2026: doravirine/islatravir 

Merck is awaiting an FDA decision for its once-daily, fixed-dose combination of doravirine/islatravir (100 mg/0.25 mg), a non-integrase-strand-transfer inhibitor (INSTI)-based antiretroviral regimen for the treatment of human-immunodeficiency virus-1 (HIV-1) infection. In a Phase 3 trial (NCT05705349) doravirine/islatravir was non-inferior to once-daily bictegravir/emtricitabine/tenofovir alafenamide (Biktarvy), with 91.8% versus 90.6% of treatment‑naïve adults achieving HIV‑1 RNA < 50 copies/mL at week 48.¹³ If approved, doravirine/islatravir will provide a single-tablet, once-daily option for treating HIV-1 infection.

April 23, 2026: isatuximab-irfc (Sarclisa) SC on-body delivery system (OBDS) 

Sanofi submitted a supplemental Biologic License Application (BLA) for subcutaneous (SC) isatuximab-irfc for the treatment of multiple myeloma. Isatuximab-irfc is a CD38-directed cytolytic antibody that is currently available under the brand name Sarclisa for IV administration for use in combination with a proteasome inhibitor and/or a thalidomide analog in patients with multiple myeloma. The open-label, Phase 3, IRAKLIA trial demonstrated SC isatuximab-irfc (1,400 mg) administered via a single-use OBDS was non-inferior to IV administered isatuximab-irfc (10 mg/kg) in patients with R/R multiple myeloma, when combined with pomalidomide and dexamethasone.¹⁴ Grade ≥ 3 treatment-emergent adverse events occurred at a rate of 81.7% and 76.1%, in the SC OBDS and IV groups respectively. In addition, infusion reactions incidences with the SC OBDS and IV administration were 1.5% and 25%, respectively, and injection site reactions occurred in 0.4% of SC OBDS injections. If approved, SC OBDS isatuximab-irfc could offer a shorter administration time compared to the IV formulation (approximately 10 minutes versus 30–60 minutes) and enhanced patient comfort with fewer injection site/infusion reactions. Isatuximab-irfc SC OBDS also has the potential for home-administration  by a health care professional.

April 23, 2026: nimodipine (GTx-104)

Grace Therapeutics is seeking FDA approval of GTx-104, an IV nanoparticle formulation of nimodipine for the treatment of aneurysmal subarachnoid hemorrhage (aSAH). The FDA granted it Fast Track and Orphan Drug designations. The open-label STRIVE-ON safety trial compared GTx-104 and oral nimodipine in patients hospitalized with aSAH. ¹⁵ The trial met its primary endpoint, reporting a 19% reduction in the incidence of clinically significant hypotension with GTx-104 compared to oral nimodipine (28% versus 35%, respectively). In addition, 29% more patients who received GTx-104 had favorable functional outcomes at 90 days compared to those who received oral nimodipine and there were fewer intensive care unit (ICU) readmissions, ICU days, and ventilator days for patients receiving GTx-104 versus oral nimodipine. Nimodipine is currently available  in oral capsule and oral liquid formulations. If approved, GTx-104 could offer an IV alternative versus nasogastric tube administration in unconscious or dysphagic patients in the hospital inpatient setting.

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References  
  1. Press release: https://www.astrazeneca.com/media-centre/press-releases/2025/baxdrostat-demonstrated-statistically-significant-clinically-meaningful-reduction-sbp-patients-hard-control-hypertension-baxhtn-phase-iii-trial.html 
  2.  Press release: https://www.astrazeneca.com/media-centre/press-releases/2025/bax24-phase-iii-trial-full-results.html 
  3.  ATOMIC-Meso trial: https://pmc.ncbi.nlm.nih.gov/articles/PMC10870227 
  4.  ASCO abstract: https://www.asco.org/abstracts-presentations/244202 
  5.  NCT05471843 Abstract: https://meetings-api.hematology.org/api/abstract/vmpreview/296316 
  6.  NCT04979806 trial: https://clinicaltrials.gov/study/NCT04979806?tab=history&a=14#version-content-panel 
  7.  NCT04251026 trial: https://www.nejm.org/doi/10.1056/NEJMoa2508681?url_ver=Z39.88-2003 
  8.  Press release: https://www.globenewswire.com/news-release/2024/09/03/2939656/0/en/Denali-Therapeutics-Announces-Successful-Meeting-with-the-FDA-and-Plans-to-File-for-Accelerated-Approval-of-Tividenofusp-Alfa-DNL310-for-the-Treatment-of-MPS-II-Hunter-Syndrome.html 
  9.  Press release: https://investors.denalitherapeutics.com/news-releases/news-release-details/denali-therapeutics-presents-enzyme-transportvehicletm-progress 
  10.  PRECISION-T trial: https://pubmed.ncbi.nlm.nih.gov/41385341/ 
  11.  ATTAIN-1 trial: https://www.nejm.org/doi/full/10.1056/NEJMoa2511774 
  12.  Press release: https://www.globenewswire.com/news-release/2025/11/07/3183988/0/en/Replimune-Presents-Late-Breaking-Abstract-and-Additional-Posters-on-RP1-at-40th-Annual-Meeting-of-the-Society-for-the-Immunotherapy-of-Cancer-SITC-2025.html 
  13.  NCT05705349 trial: https://www.thelancet.com/journals/lanhiv/article/PIIS2352-3018(26)00033-0/fulltext 
  14.  IRAKLIA trial: https://ascopubs.org/doi/10.1200/JCO-25-00744?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed 
  15.  Press release: https://www.gracetx.com/investors/news-events/press-releases/detail/291/grace-therapeutics-announces-u-s-food-and-drug-administration-acceptance-for-review-of-new-drug-application-for-gtx-104