Shots:
- Innovation peaked in December 2025, as the FDA delivered a wave of approvals spanning cardiometabolic disease, infectious disease, respiratory immunology, rare hematologic disorders, and acute cardiovascular and neurologic conditions highlighting both scientific breadth and clinical precision
- Seven notable therapies crossed the regulatory finish line, including LIB Therapeutics’ Lerochol for LDL lowering, Innoviva’s Nuzolvence for gonorrhea, GSK’s Exdensur for asthma with eosinophilic phenotype, Cytokinetics’ Myqorzo for oHCM, and Omeros’ Yartemlea for TA-TMA, alongside novel delivery innovations such as intranasal Cardamyst and Vanda’s Nereus for motion-induced vomiting
- December 2025 closed the year with strong regulatory momentum, reinforcing a clear trend toward mechanism-driven therapies, patient-centric dosing, and solutions for both high-prevalence and underserved rare diseases setting a robust foundation for launches and global expansions in 2026
Company: LIB Therapeutics
Product: Lerochol
Active Ingredient: Lerodalcibep-liga
Disease: Elevated LDL Cholesterol with Hypercholesterolemia
Date: Dec 12, 2025
Shots:
- The US FDA has approved Lerochol as an LDL-C lowering therapy for adults with hypercholesterolemia, including HeFH; US launch as a PFS is expected in spring 2026, with an autoinjector later in 2026, while EMA approval is anticipated in Jun 2026 alongside additional global regulatory filings
- Approval was supported by the global P-III (LIBerate) trial, enrolling over 2,900 CVD patients and without CVD at very and high risk of developing CVD, including HeFH; QM lerodalcibep for up to 52 wks. and over 2400 pts continued with a 72-wk extension. Lerochol achieved sustained LDL-C reductions of ~60% and was generally well tolerated
- Lerochol is a third-generation PCSK9 inhibitor with an 11-kDa polypeptide aka adnectin with high-affinity subnanomolar PCSK9 binding fused to human serum albumin to extend plasma half-life.
Company: Innoviva Specialty Therapeutics
Product: Nuzolvence
Active Ingredient: Zoliflodacin
Disease: Uncomplicated Urogenital Gonorrhea
Date: Dec 12, 2025
Shots:
- The US FDA has approved Nuzolvence for the treatment of uncomplicated urogenital gonorrhea in pts (age ≥12yrs.; Wt.≥35 kg), with US commercialization planned for the H2’26
- Approval was based on a pivotal P-III multinational trial (n=930) evaluating a single 3g oral dose of Nuzolvence vs ceftriaxone (500 mg IM) + azithromycin (1 g oral) for uncomplicated urogenital gonorrhea, which demonstrated non-inferiority, comparable tolerability with no serious adverse events, and was conducted across 16 sites in five high-prevalence countries
- Nuzolvence (single-dose oral) is a spiropyrimidinetrione antibiotic targeting bacterial type II topoisomerase, developed through a not-for-profit collaboration with GARDP, which sponsored and led the P-III trial supporting FDA approval
Company: Aptar
Product: Cardamyst
Active Ingredient: Etripamil
Disease: Paroxysmal Supraventricular Tachycardia
Date: Dec 12, 2025
Shots:
- The US FDA has approved Aptar’s Cardamyst (Etripamil), developed by Milestone Pharmaceuticals, for the conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults
- Cardamyst (Intranasal) is a Bidose (BDS) Liquid Nasal Spray System
- Aptar collaborated with Milestone Pharmaceuticals to design a custom polypropylene container closure system with an integrated cap for Cardamyst
Company: GSK
Product: Exdensur
Active Ingredient: Depemokimab-ulaa
Disease: Asthma with Eosinophilic Phenotype
Date: Dec 16, 2025
Shots:
- FDA has approved GSK’s Exdensur as an add-on maintenance therapy for asthma pts (≥12yrs.) with an eosinophilic phenotype following MHRA’s approval for both asthma & CRSwNP; regulatory review is ongoing in EU, Japan & China
- Approval was based on the P-III trials: SWIFT-1 (n=382) & SWIFT-2 (n=380) assessing Exdensur vs PBO, both in addition to SoC, which showed twice yearly Exdensur reduced annualised asthma exacerbations, achieving 58% & 48% reductions, respectively, over 52wks.
- Exdensur also reduced hospitalisation or emergency department-treated exacerbations vs PBO (SWIFT-1: 1% vs 8% SWIFT-2: 4% vs 10%), with a pooled analysis showing a 72% decrease in annualised hospitalization-requiring events over 52wks.
Company: Cytokinetics
Product: Myqorzo
Active Ingredient: Aficamten
Disease: Obstructive Hypertrophic Cardiomyopathy
Date: Dec 19, 2025
Shots:
- The US FDA has approved Cytokinetics’ Myqorzo (5, 10, 15 & 20mg) for the treatment of adults with symptomatic oHCM, available in second half of Jan 2026 through a restricted program via REMS
- Approval was based on P-III (SEQUOIA-HCM) trial of Myqorzo, showing improved exercise capacity vs PBO at 24wks., with increased peak oxygen uptake by 1.8 vs 0 ml/kg/min, consistent across all subgroups & in pts with or without background beta-blocker therapy. Results were published in The NEJM
- Myqorzo is also being evaluated in the P-III (ACACIA-HCM) trial in pts with non-obstructive HCM (nHCM) & CEDAR-HCM, in a pediatric population with oHCM
Company: Omeros
Product: Yartemlea
Active Ingredient: Narsoplimab-wuug
Disease: HSCT-Associated Thrombotic Microangiopathy
Date: Dec 23, 2025
Shots:
- The US FDA has approved Yartemlea for the treatment of HSCT-Associated Thrombotic Microangiopathy, with US launch planned for Jan 2026. MAA is under the EMA’s review, with a decision expected in mid-2026
- Approval was based on the TA-TMA Study (n=28) plus EAP (N=221 adults & pediatric pts), where 19 pts (13 adult & 6 pediatric) in the EAP had evaluable patient-level response data
- Trials showed Yartemlea improved TMA complete response, with CR in 17/28 TA-TMA Study pts & 13/19 EAP pts, plus showed 73% & 74% 100-day survival, respectively. In the peer-reviewed publications, Yartemlea was associated with 3-4x lowered mortality & higher 1yr. survival even in refractory high-risk pts
Company: Vanda Pharmaceuticals
Product: Nereus
Active Ingredient: Tradipitant
Disease: Vomiting Induced by Motion
Date: Dec 30, 2025
Shots:
- The US FDA has granted Nereus for the prevention of vomiting induced by motion, backed by 3 trials, incl. 2 P-III real-world boat studies (Motion Syros & Motion Serifos) & 1 supporting study in pts with documented motion sickness
- In Motion Syros (n=365) & Motion Serifos (n=316), vomiting incidence was 18.3–19.5% & 10.4–18.3% vs 44.3% & 37.7% with PBO, respectively, achieving more than 50–70% risk reduction in Motion Serifos
- Additionally, Vanda is advancing tradipitant for gastroparesis & for preventing nausea & vomiting associated with GLP-1 receptor agonists
Related Post: Insights+: The US FDA New Drug Approvals in November 2025
