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FDA Decisions Expected: May 2025

Your monthly synopsis of new drugs expected to hit the market 

April 14, 2025

Drug pipeline for May 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).
May–June 2025: ustekinumab biosimilar (BAT2206)
Bio-Thera Solutions is seeking FDA approval of BAT2006, an interchangeable biosimilar candidate to the human interleukin-12 and -23 antagonist ustekinumab (Stelara). Stelara is indicated for the treatment of select patients with plaque psoriasis, psoriatic arthritis, Crohn’s disease or ulcerative colitis. If approved, BAT2206 will be the eighth biosimilar to Stelara approved by the FDA and the second that will be interchangeable with the reference product.
May 5, 2025: pegzilarginase
Immedica submitted pegzilarginase for FDA-approval for the treatment of arginase 1 deficiency (ARG1-D), a rare, genetic, metabolic urea cycle disorder. ARG1-D is characterized by a lack of functional arginase enzyme in the liver and red blood cells that leads to high levels of arginine in the blood and cerebrospinal fluid. Excess accumulation of ammonia in the blood can also occur. If left untreated, ARG1-D leads to muscle spasticity, gait disorders, developmental delay, failure to thrive and seizures. The current management approach for ARG1-D consists of a life-long severe protein restricted diet to reduce plasma arginine (pArg) levels and supplementation of essential amino acids. Ammonia scavenger medications (e.g., sodium phenylacetate, sodium benzoate) to clear accumulated ammonia from the blood may also be used. Pegzilarginase is a recombinant cobalt-substituted and pegylated human arginase 1 (ARG1) enzyme that lowers levels of arginine and its toxic metabolites in the plasma. If approved, pegzilarginase will be the first medication that reduces pArg to normal levels in patients with ARG1-D. It was evaluated in the double-blind, placebo-controlled, Phase 3 PEACE trial.¹ Pegzilarginase was administered once weekly as an intravenous (IV) infusion for the first eight weeks of treatment, after which patients could switch to subcutaneous (SC) administration given at home by a health care professional. In the trial, pegzilarginase led to a 76.7% reduction in geometric mean pArg levels compared to placebo (p<0.0001) at 24 weeks. A statistically significant improvement in functional mobility was not demonstrated; however, data from the trial suggest that longer-term use of pegzilarginase may lead to clinical stabilization of patients with ARG1-D. Pegzilarginase received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations as well as a Priority Review from the FDA for the treatment of ARG1-D.

For more information, see the pegzilarginase Deep Dive in the January 2025 edition of Prime’s Quarterly Drug Pipeline.
May 27, 2025: sodium dichloroacetate (SL-1009)
Saol Therapeutics is awaiting an FDA decision for SL-1009 oral solution for the treatment of pyruvate dehydrogenase complex deficiency (PDCD). PDCD is a rare mitochondrial disorder of carbohydrate oxidation caused by mutations in the genes that encode pyruvate dehydrogenase complex that plays a role in energy production for proper cell function. PDCD is typically associated with neurologic manifestations (e.g., abnormal brain imaging, hypotonia, seizures, ataxia) and metabolic abnormalities (increased plasma pyruvate, lactic acidemia, metabolic acidosis). The age of onset and severity of disease symptoms vary widely, ranging from onset in the prenatal period or in infancy leading to death in early childhood to onset in later childhood with survival into adulthood. Carbohydrate restricted (ketogenic) diets and co-factor (e.g., thiamine) supplementation have been used to manage PDCD and may provide benefit. SL-1009 was granted Fast Track, Orphan Drug and Rare Pediatric Disease designations and a Priority Review from the FDA. SL-1009 has been evaluated in a double-blind, placebo-controlled, cross-over Phase 3 trial (NCT02616484) with a primary endpoint of daily Observer Reported Outcomes (ObsROmotor) survey tool to measure changes in motor domains and a keysecondary endpoint of reduction in plasma lactate.² However, results have not been announced publicly. A survival analysis (SL1009-02 study) that compared outcomes for SL-1009-treated patients in the Phase 3 study with an untreated natural history cohort also supported the FDA submission. If approved, SL-1009 will be the first medicine available to treat PCDC.
May 27, 2025: telisotuzumab vedotin (Teliso-V)
Abbvie is seeking Accelerated Approval of Teliso-V in adults with previously treated, locally advanced or metastatic epidermal growth factor receptor (EGFR) wild type, nonsquamous non-small cell lung cancer (NSCLC) with c-Met protein overexpression. Teliso-V is a first-in-class antibody-drug conjugate (ADC) that targets c-Met, a receptor tyrosine kinase that can be overexpressed in many solid tumors. The Phase 2 LUMINOSITY trial demonstrated that Teliso-V administered IV every two weeks resulted in an overall response rate of 28.6% and a median duration of response of 8.3 months.³ Grade 5 treatment-related adverse events occurred in two patients and included interstitial lung disease and respiratory failure. Teliso-V has a Breakthrough Therapy designation. If it was granted a Priority Review, an FDA decision could be made as early as May 27, 2025. If approved, Teliso-V will be the first treatment specifically indicated for c-Met overexpressing NSCLC.
May 30, 2025: acoltremon (AR-15512)
Acoltremon, by Alcon, is a first-in-class transient receptor potential melastatin 8 (TRPM8) agonist submitted to the FDA for the treatment of dry eye disease (DED). Top-line results from the randomized, double-masked, vehicle-controlled, Phase 3 COMET-2 and COMET-3 trials demonstrated that significantly more patients with DED who received acoltremon experienced an increase in tear production at Day 14 as evidenced by the primary endpoint measure of ≥ 10-mm increase in unanesthetized Schirmer’s score (p<0.0001 in each trial) compared to patients who received vehicle.⁴ Efficacy was observed as early as Day 1 and was maintained through Day 90 of treatment. In the clinical studies, monotherapy with acoltremon 0.003% ophthalmic solution was administered in both eyes twice daily. If approved, acoltremon will provide a unique mechanism of action for the treatment of DED.
May 30, 2025: COVID-19 vaccine (mRNA-1283)
Moderna submitted their next-generation vaccine mRNA-1283 for the prevention of Coronavirus disease-2019 (COVID-19) infection using a Priority Review voucher. The randomized, observer-blind, active-controlled, Phase 3 NextCOVE trial (n=11,417) compared mRNA-1283 with the bivalent COVID-19 vaccine mRNA-1273 (Spikevax).⁵ Single doses of the vaccines were administered intramuscularly as 10 µg and 50 µg, respectively. Interim results demonstrated that the relative vaccine efficacy (rVE) of mRNA-1283 was non-inferior compared to mRNA-1273. In the trial, mRNA-1283 was well tolerated. No events of myocarditis or pericarditis were reported.
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