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FDA Decisions Expected: June 2025

Your monthly synopsis of new drugs expected to hit the market 

May 21, 2025

Drug pipeline for June 2025

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).
June 2025: ranibizumab (biosimilar to Genentech's Lucentis) 
Stada and Xbrane resubmitted their investigational biosimilar to ranibizumab (Lucentis) to the FDA for review. If approved, it will be the third biosimilar for Lucentis in the U.S. Lucentis is a vascular endothelial growth factor (VEGF) inhibitor for intravitreal injection that is indicated for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy (DR) and myopic choroidal neovascularization (mCNV). This is the second review of the biosimilar candidate after the FDA issued a complete response letter (CRL) in April 2024 concerning the reference standard and manufacturing site inspections. 
June 2025: garadacimab 
CSL Limited is awaiting FDA decision of garadacimab for prophylactic treatment of hereditary angioedema (HAE). Garadacimab is a first-in-class recombinant monoclonal antibody that targets activated factor XII (FXII). It was granted Fast Track and Orphan Drug designations by the FDA. This is the second review for garadacimab by the FDA, after the agency issued a CRL requesting information pertaining to the manufacturing process. Garadacimab was evaluated in the double-blind, parallel-group VANGUARD trial in patients ≥ 12 years of age with HAE.¹  During a six-month treatment period, once monthly subcutaneous (SC) doses of garadacimab led to a significant decrease in the primary efficacy endpoint of mean number of investigator-confirmed HAE attacks per month compared to placebo (0.27 versus 2.01, respectively; p<0.0001). No increase in the risk of bleeding or thromboembolic events was observed. If approved, garadacimab will be available for administration by the patient or caregiver.

For more information, see the garadacimab Deep Dive in the July 2024 edition of Prime’s Quarterly Drug Pipeline 
Jun–Oct 2025: sodium chlorite (NP001)
Neuvivo submitted NP001 to the FDA for the treatment of patients with amyotrophic lateral sclerosis (ALS), a neurodegenerative disease characterized by progressive loss of voluntary muscle function. Death in patients with ALS occurs within 2 to 5 years after diagnosis, usually due to respiratory failure. NP001 regulates pro- and anti-inflammatory processes of the innate immune system and may help slow the progression of ALS. A randomized, double-blind, placebo-controlled Phase 2a trial (NCT01281631) reported that NP001 did not lead to a statistically significant decline in ALS progression as measured by ALS Functional Rating Scale-Revised (ALSFRS-R) in the overall study population over a six-month treatment period.² However, further analysis published in 2023 revealed a 64% slower rate of decline in respiratory vital capacity (VC), a predictor of ALS survival, among a subset of patients ≤ 65 years of age with a high plasma C-reactive protein (CRP ≥ 1.13 mg/L) level who received NP001 at a dose of 2 mg/kg.³ In addition, a blinded, placebo-controlled, retrospective analysis with up to 11 years of data from the six-month Phase 2a (NCT01281631) and Phase 2b (NCT02794857) studies found NP001 extended patient survival by 4.8 months in the total study population and by 10.8 months in a subset of patients ≤ 65 years of age.⁴ If approved, NP001 will provide a new mechanism of action for the treatment of ALS, a condition with considerable unmet need. The FDA granted NP001 Fast Track and Orphan Drug designations. The product is also eligible for Accelerated Approval and Priority Review; if granted FDA decision could be made as early as June 2025.
June 10, 2025: clesrovimab
Merck submitted clesrovimab to the FDA for respiratory syncytial virus (RSV) disease prevention in infants during their first RSV season. If approved, clesrovimab will be the third monoclonal antibody available to prevent RSV infection in pediatric patients; the others are palivizumab (Synagis) and nirsevimab-alip (Beyfortus), which are approved in infants and young children (≤ 24 months of age) who are at risk for serious RSV-related severe lower respiratory tract disease (LRTD). In the double-blind, Phase 2b/3 CLEVER trial, a single intramuscular (IM) dose of clesrovimab reduced the primary efficacy endpoint of incidence of RSV-associated medically attended lower respiratory infections (MALRI) by 60.4% (p<0.001) compared to placebo in healthy preterm and full-term infants during their first RSV season. In addition, top-line results from the Phase 3, partially blinded, SMART trial reported comparable rates of RSV-associated MALRI and safety profiles between one dose of clesrovimab and doses of palivizumab (Synagis) administered in accordance with national or local guidelines or professional society recommendations in infants and children at increased risk for severe RSV disease.⁵ Unlike Beyfortus, the dosage for clesrovimab is the same for all patients during the first RSV season, regardless of their body weight. Merck anticipates clesrovimab will be available for the 2025-2026 RSV season if FDA approval is granted.

For more information, see the clesrovimab Deep Dive in the January 2025 edition of Prime’s Quarterly Drug Pipeline. 
June 13, 2025: mitomycin (UGN-102)
UGN-102, a reverse thermal hydrogel containing mitomycin for intravesical solution, is under 505(b)(2) NDA review by the FDA for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). In clinical trials, UGN-102 was administered as six weekly intravesical instillations.⁶ The open-label, Phase 3 ATLAS trial compared UGN-102 to transurethral resection of bladder tumor (TURBT) in patients with newly diagnosed or recurrent LG-IR-NMIBC.⁷ The study demonstrated among patients who achieved a complete response at three months, 79.7% in the UGN-102 group were event-free at 12 months compared to 67.7% in the TURBT group. Similarly, in the single-arm, Phase 3 ENVISION trial, 79.6% of patients achieved a complete response with UGN-102 at three months, of whom 82.3% remained event-free at 12 months. If approved, UGN-102 will provide a minimally invasive, non-surgical, out-patient option for treating LG-IR-NMIBC. In addition, top-line results from a small, single-arm, Phase 3b study (NCT05136898) suggest feasibility of UGN-102 for in-home administration by a health care professional.⁸
June 17, 2025: sebetralstat
Kalvista is awaiting the FDA’s decision for their oral plasma kallikrein inhibitor sebetralstat for on-demand treatment of hereditary angioedema (HAE) in patients ≥ 12 years of age. FDA granted sebetralstat Fast Track and Orphan Drug designations. Published results from the Phase 3, double-blind, three-way crossover KONFIDENT trial reported that the time to the beginning of symptom relief was significantly faster with sebetralstat 300 mg and sebetralstat 600 mg compared to placebo (median, 1.61 hours, 1.79 hours, and 6.72 hours, respectively; p≤0.001 for both doses).⁹ If approved, sebetralstat will be the second plasma kallikrein inhibitor indicated to treat acute attacks of HAE. Moreover, it will be the first oral agent for on-demand use for HAE, providing an important option that will minimize treatment burden and may prevent treatment delays.

For more information, see the sebetralstat Deep Dive in the January 2025 edition of Prime’s Quarterly Drug Pipeline. 
June 23, 2025: taletrectinib
Nuvation Bio is awaiting FDA decision for taletrectinib for the treatment of locally advanced or metastatic ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC) when no comparable or satisfactory alternative therapy options are available. It is estimated that ROS1 gene rearrangements occur in about 1% to 2% of patients with NSCLC. Taletrectinib is a potent, central nervous system-active, selective, next-generation ROS1 tyrosine kinase inhibitor (TKI). The FDA granted it Breakthrough Therapy and Orphan Drug designations as well as a Priority Review for NSCLC. Pooled data from the ongoing, single-arm, Phase 2 TRUST-I and TRUST-II trials demonstrated high and durable overall response rates (ORR) with once daily oral doses of taletrectinib in patients with locally advanced or metastatic ROS+ NSCLC after a median follow-up of about 21 months.¹⁰ The confirmed ORR (primary endpoint) was 55.8% in patients who received prior TKI therapy and 88.8% in those who were naïve to TKIs. In addition, in TKI-naive patients, the median duration of response (DOR) and median progression-free survival (PFS) were 44.2 months and 45.6 months, respectively. Among TKI-pretreated patients, the median DOR and median PFS were 16.6 months and 9.7 months, respectively. Notably, an estimated one-third of newly diagnosed patients with metastatic ROS1+ NSCLC have intracranial involvement. In the Phase 2 trials, taletrectinib resulted in an intracranial confirmed ORR of 76.5% in TKI-naïve patients and 65.6% in TKI-pretreated patients. Taletrectinib had a favorable tolerability and safety profile. If approved, taletrectinib will provide a third TKI option for ROS1+ NSCLC, following crizotinib (Xalkori) and entrectinib (Rozlytrek).
June 28, 2025: oxylanthanum carbonate
Oxylanthanum carbonate (OLC) by Unicycive contains lanthanum, the same active moiety as the approved phosphate binder lanthanum carbonate (Fosrenol), and forms the same insoluble phosphate complex, lanthanum phosphate, in the gastrointestinal tract. Unicycive’s proprietary nanoparticle formulation of OLC allows for the same binding capacity as lanthanum carbonate in a smaller tablet that can be easily swallowed compared to the currently available large tablets of lanthanum carbonate that must be chewed. Unicycive is pursuing FDA approval of OLC via the 505(b)(2) regulatory pathway for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD). A Phase 1, open-label, two-way crossover study demonstrated bioequivalence between OLC and lanthanum carbonate in healthy individuals.¹¹ In addition, a Phase 2, multidose trial (NCT06218290) reported OLC compared to pre-trial phosphate binders reduced pill burden by 50% (3 tablets versus 6 tablets, respectively), improved adherence to therapy (70% versus 58%, respectively) and increased patient satisfaction (98% versus 38%, respectively), in patients with CKD.¹²
June 30, 2025: copper histidinate (CUTX-101)
CUTX-101 is a copper replacement therapy designed to maintain serum copper levels that is under Priority Review by the FDA for the treatment of Menkes disease, a rare, fatal, congenital, X-linked disorder associated with improper absorption of copper from the diet. Characteristic features of Menkes disease include hypotonia, seizures, failure to thrive, progressive neurodegeneration and connective tissue dysfunction. Death usually occurs by 3 years of age. Investigational parenteral copper replacement therapy has been used in clinical trials or expanded access/compassionate use programs to treat Menkes disease. Studies suggest that initiating parenteral copper replacement therapy by four weeks of age may improve clinical outcomes. If approved, CUTX-101 will be the first medicine available for Menkes disease. Clinical data were combined from two long-term, open-label, single-arm, single-site studies, including a Phase 1/2 study (NCT00001262) and a Phase 3 study (NCT00811785), that evaluated CUTX-101 in patients with severe ATP7A genotype Menkes disease.¹³ Analysis revealed a significant improvement in the primary endpoint of overall survival (OS) among patients who received early treatment (started within four weeks of birth) with CUTX-101 compared to the historical control group (median OS, 177.1 months verses 16.1 months, respectively; p<0.0001). As a secondary endpoint, a significant improvement in OS was also reported among patients who started CUTX-101 treatment later (after four weeks from birth) compared to the historical control group (median OS, 62.4 versus 17.6 months, respectively; p<0.0001). In addition, the Phase 1/2 trial reported significant improvements in gross motor, fine motor/adaptive, personal-social and language neurodevelopment in early treatment subjects who initiated CUTX-101 prior to onset of symptoms.

For more information, see the copper histidinate Deep Dive in the April 2025 edition of Prime’s  Quarterly Drug Pipeline. 

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References  

  1. VANGUARD trial: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(23)00350-1/abstract

  2. NCT01281631 trial published June 2015: https://www.neurology.org/doi/full/10.1212/NXI.0000000000000100

  3. NCT01281631 trial published 2023: https://www.mdpi.com/2073-4409/12/7/1031

  4. Long-term data: https://www.mdpi.com/2227-9059/12/10/2367

  5. Press release: https://www.businesswire.com/news/home/20241017875997/en/Mercks-Clesrovimab-MK-1654-an-Investigational-Respiratory-Syncytial-Virus-RSV-Preventative-Monoclonal-Antibody-Significantly-Reduced-Incidence-of-RSV-Disease-and-Hospitalization-in-Healthy-Preterm-and-Full-term-Infants

  6. Investor presentation: https://investors.urogen.com/static-files/fba40caa-c888-42fa-9f09-545ac8346efd

  7. ATLAS & ENVISION trials: https://meetings.asco.org/abstracts-presentations/242879

  8. Press release: https://www.businesswire.com/news/home/20230215005534/en/Investigational-Therapy-UGN-102-May-Be-Delivered-at-Home-for-the-Treatment-of-Bladder-Cancer

  9. KONFIDENT trial: https://www.nejm.org/doi/full/10.1056/NEJMoa2314192

  10. ESMO presentation: https://nuvationbio.com/wp-content/uploads/2024/11/ESMO-2024-Pooled-TRUST-I-II-Poster_Final_Revised.pdf

  11. NCT06218290 trial: https://www.clinicaltherapeutics.com/article/S0149-2918(24)00351-5/fulltext

  12. Press release: https://ir.unicycive.com/news/detail/98/unicycive-presents-new-patient-level-data-underscoring

  13. NCT00001262 and NCT00811785 trials: https://publications.aap.org/pediatrics/article/149/1%20Meeting%20Abstracts%20February%202022/252/185910/Copper-Histidinate-Treatment-for-Menkes-Disease?autologincheck=redirected