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FDA Decisions Expected: September 2025

Your monthly synopsis of new drugs expected to hit the market 

August 14, 2025

Drug pipeline for September 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).
September–October 2025: denosumab (RGB-14-P)  
Gedeon Richter is awaiting FDA approval of RGB-14-P, a biosimilar candidate for the RANK ligand (RANKL) inhibitor denosumab. The denosumab reference products are Prolia and Xgeva, by Amgen. Prolia is approved to treat osteoporosis or increase bone mass in select men and women who are at high risk for bone fracture. Xgeva is indicated for use in certain patients with cancers affecting the bone. If approved, RGB-14-P will join multiple other denosumab biosimilars in the U.S.  
Sept. 14, 2025: bumetanide nasal spray  
The FDA is reviewing bumetanide nasal spray by Cortasis under the 505(b)(2) new drug application (NDA) pathway for the treatment of edema related to congestive heart failure, liver disease and kidney disease. Bumetanide is currently available as oral tablets and as a solution for intravenous (IV) or intramuscular (IM) administration for use when oral administration is not practical or gastrointestinal (GI) absorption is impaired. In the RSQ-777-02 open-label, cross-over study conducted in 68 healthy adults, bumetanide nasal spray led to a similar amount of urine output as the oral and IV formulations.¹ Absorption of the nasal and IV formulations were less variable compared to the oral tablets. The nasal spray was well tolerated, with no significant nasal irritation reported. If approved, bumetanide nasal spray could provide a self-administered, outpatient alternative to parenteral formulations in patients with impaired GI absorption or difficulty swallowing.  
Sept. 22, 2025: apitegromab  
Scholar Rock is awaiting FDA decision for their selective latent myostatin inhibitor apitegromab for the treatment of spinal muscular dystrophy (SMA). The FDA granted apitegromab a Priority Review as well as Fast Track, Orphan Drug and Rare Pediatric Drug designations. In the double-blind, Phase 3 SAPPHIRE trial, apitegromab added onto standard of care (SOC) led to a statistically significant and clinically meaningful improvement in the primary endpoint of motor function at week 52 compared to placebo as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory patients with type 2 or type 3 SMA. Approximately 30.4% of patients who received apitegromab had >3 point improvement in HFMSE compared to 12.5% of patients who received placebo.² If approved, apitegromab will provide a new mechanism of action to treat SMA. Notably, the Institute for Clinical and Economic Review (ICER) released a revised evidence report assessing the comparative clinical effectiveness and value of treatments for SMA, including apitegromab. ICER stated that the addition of apitegromab to SOC in patients 2–12 years of age with type 2 or type 3 SMA likely provides comparable or incremental benefits compared with no additional therapy, but that there is some possibility of substantial benefit with long-term use as well as some possibility of net harm. 

For more information, see the apitegromab Deep Dive in the July 2025 edition of Prime’s Quarterly Drug Pipeline 
Sept. 23, 2025: pembrolizumab/berahyaluronidase (Keytruda SC)  
Merck submitted a combination product containing pembrolizumab and berahyaluronidase alfa as a subcutaneous (SC) version of pembrolizumab (Keytruda) for approval across all previously approved solid tumor indications for Keytruda. An open-label Phase 3 trial (NCT05722015) demonstrated noninferior overall exposure (AUC0-6 weeks) and steady-state trough concentration (Ctrough) of pembrolizumab with pembrolizumab/berahyaluronidase alfa administered SC over a median time of two minutes (range, 1-12 minutes) compared to IV Keytruda administered over about 30 minutes; both formulations were given every 6 weeks.³ In the trial, both products were administered with chemotherapy for the first-line treatment of adults with metastatic non-small cell lung cancer (NSCLC) and demonstrated similar efficacy and safety. If approved, pembrolizumab/berahyaluronidase alfa will provide a new SC checkpoint inhibitor option that may increase access and decrease treatment burden compared to IV Keytruda. It will be the second SC-administered programmed death receptor-1 (PD-1)-blocking antibody, following nivolumab/hyaluronidase (Opdivo Qvantig). 
Sept. 25, 2025: paltusotine  
Paltusotine is a somatostatin receptor ligand (SSL) by Crinetics under FDA review for the treatment and long-term maintenance therapy of acromegaly in adults. In Phase 3 trials, paltusotine was administered orally once daily. In the double-blind, placebo-controlled PATHFNDR-1 trial, based in interim data, paltusotine maintained mean insulin-like growth factor 1 (IGF-1) levels (≤1.0 x upper limit of normal [UNL]) and symptom control at week 96 in patients with acromegaly who were switched from monthly injectable medications.⁴ Interim data of the double-blind, placebo-controlled PATHFNDR-2 trial revealed patients who had received placebo during the randomized controlled phase and switched to paltusotine in the open-label extension period experienced sustained reductions in IGF-1, with a mean change from baseline of –0.81 × ULN at Week 84. Paltusotine received Orphan Drug designation from the FDA. If approved, will be the second oral treatment for acromegaly, following Mycapssa (octreotide).   
Sept. 28, 2025: tolebrutinib  
Sanofi is awaiting FDA approval of their oral Bruton’s tyrosine kinase (BTK) inhibitor tolebrutinib for the treatment of non-relapsing secondary progressive multiple sclerosis (SPMS) and to slow disability accumulation independent of relapse activity.  The FDA granted Breakthrough Therapy designation and a Priority Review for tolebrutinib. In the double-blind, Phase 3 HERCULES trial, the primary endpoint of confirmed disability progression sustained for at least 6 months was observed in a smaller proportion of adult patients with non-relapsing SPMS in the tolebrutinib group compared to the placebo group (22.6% versus 30.7%, respectively; p=0.003).⁵ If approved, tolebrutinib will be the first BTK inhibitor for the treatment of MS and the first therapy specifically approved for non-relapsing SPMS. Tolebrutinib is differentiated by its ability to penetrate the brain to target B cells compared to existing medicines that target peripheral B and T cells. 

For more information, see the tolebrutinib Deep Dive in the July 2025 edition of Prime’s Quarterly Drug Pipeline 
Sept. 30, 2025: copper histindinate (CUTX-101)  
CUTX-101 is a copper replacement therapy by Fortress designed to maintain serum copper levels. It is awaiting FDA approval for the treatment of Menkes disease, a rare, congenital, X-linked, metabolic disorder characterized by hypotonia, seizures, failure to thrive, progressive neurodegeneration, connective tissue dysfunction and life expectancy of about 3 years. CUTX-101 was granted Breakthrough Therapy, Fast Track Designation, Rare Pediatric Disease Designation and Orphan Drug designations and a Priority Review. If approved, CUTX-101 will be the first medicine available for Menkes disease. In clinical trials, it was associated with significant improvement in survival and neurological outcomes compared to an untreated historical control group.⁶ Fortress initially announced an FDA target action date of June 30, 2025; however, this was later revised to Sept. 30, 2025. 

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