Pharmaceutics and Drug Design Commons^™

2025 Novel Drug Approvals, Katherine Ghattas Pharmd, Amanda Rawa Pharmd

Transformative Medicine

The year 2025 marked another strong chapter in pharmaceutical innovation, with multiple novel therapies gaining approval from the U.S. Food and Drug Administration (FDA). These approvals reflect continued progress in addressing unmet medical needs across a wide range of disease states. This article reviews the FDA’s 2025 novel drug approvals, spotlighting a few therapeutic advancements and emerging trends. A comprehensive table of all approved agents is provided, along with a focused discussion of four particularly impactful therapies: donidalorsen (Dawnzera), taletrectinib (Ibtrozi), delgocitinib (Anzupgo), gepotidacin (Blujepa), etripamil (Cardamyst). Each of these agents represents a step forward in its respective field and …

Efficacy And Safety Of Stem Cell-Based Interventions For Burn Wound Healing: A Systematic Review And Meta-Analysis Of Pre-Clinical And Early Clinical Evidence, Prayuth Poowaruttanawiwit, Patsanan Kulla, Naveeya Pakpoomkamonlert

The Thai Journal of Pharmaceutical Sciences

Background: Burn injuries remain a major therapeutic challenge, with conventional treatments often failing to achieve complete functional skin regeneration. Stem cell-based interventions, including mesenchymal stem cells (MSCs) and exosome derivatives, have demonstrated regenerative and immunomodulatory potential, yet their translation to clinical practice remains limited.
Objectives: To systematically evaluate the efficacy and safety of stem cell-based therapies for burn wound healing across pre-clinical and clinical studies.
Materials and Methods: Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses 2020 guidelines, four databases (PubMed, Embase, Scopus, and Cochrane) were searched through April 2025. Eligible studies included randomized controlled trials and controlled pre-clinical …

Development Of Aqueous Dispersions Of Zein–Inulin Nanoparticles: Influence Of Formulation Variables On Physical Properties, Wanarat Sayamol, Supakarn Chamni, Jittima Chatchawalsaisin

The Thai Journal of Pharmaceutical Sciences

Background: Zein-based nanoparticles exhibit considerable potential for pharmaceutical and food applications. Incorporating inulin, a prebiotic polysaccharide, can improve their functional performance. Owing to the distinct solubilities of zein and inulin in aqueous ethanol, zein– inulin nanoparticles can be prepared through precipitation using their respective anti-solvents.
Objectives: This study aimed to systematically develop zein–inulin nanoparticles using the antisolvent precipitation method, with a focus on investigating the effects of formulation variables on particle characteristics and dispersion properties.
Materials and Methods: Zein–inulin nanoparticles were prepared using an anti-solvent precipitation method. Zein and inulin were separately dissolved in aqueous ethanol and water, respectively. The …

Receptor-Mediated Drug Delivery: Redefining Targeted Drug Conjugates In Oncology, Keon Niles Jafari, Charlene Chai, Shelby Kim, Kamaljit Kaur

Pharmacy Faculty Articles and Research

Targeted drug delivery (TDD), specifically through targeting ligand–drug conjugates, has reshaped oncology by enabling selective delivery of cytotoxic payloads to cancer cells while minimizing uptake by normal tissues. A key approach relies on exploiting overexpressed cell surface receptors (CSRs) to enable selective uptake of drug conjugates via receptor-mediated endocytosis. This review delineates four clinically validated CSRs (HER2, Trop-2, Nectin-4, and SSTR2) with several FDA-approved drug conjugates. Furthermore, emerging CSRs (EGFR, DLL3, and keratin 1) that may support next-generation TDD platforms for cancer treatment are also highlighted. We discuss how CSR type, density on cancer cells, and its mechanism of endocytosis, …

Gadolinium Nanoparticles: Emerging Platforms Beyond Imaging For Drug Delivery And Theranostics, Amir Nasrolahi Shirazi, Rajesh Vadlapatla, Ajoy Koomer, Heyam Zayed, Paris Marabut, Keykavous Parang

Pharmacy Faculty Articles and Research

Gadolinium nanoparticles (GdNPs) have gained increasing attention as multifunctional metal-based nanoplatforms that extend far beyond their traditional use as magnetic resonance imaging (MRI) contrast agents. Their specific magnetic properties, tunable physicochemical features, and tunable biocompatibilities with biocompatible coatings give them great potential as drug delivery and theranostic applications. They offer greater stability, lower systemic toxicity, and more surface modification options compared to molecular gadolinium chelates. The functionalized GdNPs not only show excellent properties as drug carriers for their specific indications but also serve as agents in various imaging modalities with superior therapeutic efficacy by means of radio sensitization and magnetically …

Microbiome-Responsive Hydrogels: From Biological Cues To Smart Biomaterials, Rajesh Vadlapatla, Amir Nasrolahi Shirazi, Ajoy Koomer, Judy Weng, Matthew Ernest Ghilarducci, Alai Qudus, Keykavous Parang

Pharmacy Faculty Articles and Research

Background: Stimuli-responsive hydrogels (SRHs) are smart polymeric materials that undergo reversible physicochemical changes in response to abiotic cues and externally applied fields, enabling applications in drug delivery, wound healing, and tissue engineering. However, they exhibit limited biological specificity and do not adequately reflect the dynamic, disease-relevant complexity of native tissue microenvironments. Microbe-colonized tissues display distinctive biochemical features driven, shaped by microbial metabolism, including localized pH gradients, short-chain fatty acid production, secretion of quorum-sensing molecules, biofilm formation, and expression of specialized enzymes. These endogenous, spatiotemporally regulated signals are closely linked to host physiology and pathology but remain underutilized in hydrogel …

Evidentiary Standards For Newly Approved Antibiotics For Uncomplicated Urinary Tract Infections, Rosa Rodriguez-Monguio, Enrique Seoane-Vazquez, John H. Powers Iii

Pharmacy Faculty Articles and Research

Importance: Uncomplicated urinary tract infections (uUTIs) are among the most common bacterial infections and are typically treated with existing oral antibiotics. In 2024–2025, the FDA approved two new oral agents, sulopenem etzadroxil/probenecid and gepotidacin, via expedited review pathways, for the treatment of uUTIs. Objective: To assess the clinical significance and regulatory evidence supporting FDA approval of sulopenem/probenecid and gepotidacin for uUTI, and to analyze the adherence of pivotal phase III trials to regulatory standards for approval and FDA guidelines. Design, Setting, and Participants: Comparative descriptive analysis of publicly available data from phase 3 randomized, double-blind, active-controlled clinical …

Optimization Of A Kcc2 Small Molecule Agonist And The Development Of An In-Silico Model Of Intermolecular Dynamics For The Rational Design Of Therapeutics Against Opioid Use Disorder, William H.D. Ho, Alfred Amendolara, Kenyon Mitchell, Jaden Miner, Braxton Bingham, Andrew Payne

Annual Research Symposium

Roseman University Research Symposium

Wednesday, February 18, 2026 | 3:00 – 6:00 pm (local time)

Tumor Targeting With Peptide-Drug Conjugates: Showcasing Key Progress And Hurdles, Keykavous Parang, Thuy Do, Clare Dinh, Dorna Davanidavari, Max Foroughi, Troy Khong, Mahsa Moazen, Amir Nasrolahi Shirazi

Pharmacy Faculty Articles and Research

Peptide-drug conjugates (PDCs) are modular, targeted therapeutics composed of a homing peptide linked to a cytotoxic or modulating drug payload via a cleavable/non-cleavable linker. PDCs utilize peptide targeting to enhance the delivery of potent drugs to tumors, providing advantages such as superior tissue penetration, reduced immunogenicity, and simpler manufacture compared to antibody-drug conjugates (ADCs). A comparison of PDCs versus ADCs highlights that PDCs’ small size (~1-3 kDa) enables deeper tumor penetration and faster clearance, whereas ADCs (~150 kDa) benefit from prolonged circulation but suffer from limited tissue diffusion. This review surveys recent advances in PDC design and application. We discuss …

Protein Allostery Probed By Ligands Across Sites, Virgil A. Woods

Dissertations, Theses, and Capstone Projects

Allostery is a pervasive regulatory principle throughout biology, yet the structural pathways by which distal inputs alter active‑site chemistry within protein structures remain incompletely defined and exploited. This dissertation uses protein tyrosine phosphatase 1B (PTP1B) as a tractable model to map those pathways with complementary experimental and computational tools. I integrate high‑resolution hydrogen-deuterium exchange mass spectrometry (HDX-MS), room-temperature crystallography, NMR, steady-state kinetics, crystallographic pseudo-ensembles, and machine-learning-guided ligand discovery. The working premise is that regulation reflects redistribution within a conformational ensemble rather than a binary switch. That orthogonal perturbations by small molecules, mutations, and protein partners can be used to both …

Impact Of Apitegromab On Motor Function In Spinal Muscular Atrophy, Jordyn Linfield, Ashley Jacob, Haylee Whyde, Owen Fox, Kaitlyn Bowden, Connor Dains, Brenna Hissong, Manoranjan D'Souza

Pharmacy and Wellness Review

Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder characterized by the progressive loss of alpha motor neurons in the anterior horn of the spinal cord, leading to muscle weakness, atrophy, and impaired motor function.¹ SMA remains an extensive and difficult-to-treat condition, with only three disease-modifying therapies currently available to patients. A new investigational agent with a unique mechanism of action, apitegromab (SRK-015), has shown promising results in recent clinical trials. The safety and efficacy of apitegromab in patients with SMA have been explored in several clinical trials, with the TOPAZ and SAPPHIRE studies being the most notable. This …

Fda Policy Shift On Clinical Efficacy Study Requirements For Monoclonal Antibody Biosimilars, Rileigh Rahrig, Madilyn Paphanchith, Allie Honigford, Daniella Egbujor, Haley Brown, Jenny Magana Figueroa, Connor Dains, Brenna Hissong, Kyle W. Parker

Pharmacy and Wellness Review

Biologic products are widely used in the treatment of various disease states, including rheumatoid arthritis, inflammatory bowel disease, cancers, and genetic diseases. These products are derived from proteins and genes produced by living organisms and are subsequently used to treat specific disease states. Due to the complexity of biological products, the costs of research and production are high. Currently, the United States approval pathway requires extensive testing for biologics to be designated as biosimilars, meaning there are no clinically meaningful differences in safety or purity of their active components. These required tests can be redundant, as biosimilars are developed using …

Evaluation Of Peptides And Peptide–Doxorubicin Conjugates Designed And Synthesized For Targeting Triple-Negative Breast Cancer Via Cell-Surface Receptors, Shih-Jing (Jane) Yao

Pharmaceutical Sciences (PhD) Dissertations

A significant problem faced in oncology is the lack of chemotherapeutic agents that are tumor site-specific, resulting in reduced therapeutic efficacy and unintended infliction of harm to surrounding healthy tissues and cells that often lead to serious side effects. Current strategies being explored to circumvent this issue aim to refine delivery of  chemotherapeutic agents specifically to tumor sites, which may be achieved via conjugation of these agents to biomarker-specific ligands. This forms the foundation of ligand-targeted drug delivery. Among different breast cancers, treatment of the triple-negative breast cancer (TNBC) subtype has been particularly challenging, largely due to the absence of …

Evaluation Of The Physical Properties And In Vitro Safety Of Chitosan-Quercus Infectoria Topical Gel, Rani Naga Rakshitha Tillapudi

Theses, Dissertations and Capstones

Due to ongoing inflammation, inadequate blood flow, oxidative stress, and reduced tissue regeneration, chronic diabetic wounds heal slowly, which is a serious health concern. It is important to examine novel strategies that tackle these issues because conventional treatments frequently fail. Therefore, this study examined the physical properties and biocompatibility of a novel topical gel formulated with chitosan and Quercus infectoria gall extract.

Over 28 days, pH, spreadability, texture, homogeneity, color, and drug concentration were assessed. UV-visible spectrophotometry and FTIR analysis were used to establish chemical stability. The MTT assay was used to test its safety in human dermal fibroblast adult …

Nanocomposite Mediated Delivery Of 5-Fluorouracil Using Silver Nanoparticles To Promote Wound Healing, Kamal Nath Kasi

Theses, Dissertations and Capstones

Chronic wounds, particularly in diabetic patients, remain a significant clinical challenge due to the complex interaction of persistent infection, chronic inflammation, and impaired cellular regeneration. Conventional drug therapies often fail to achieve effective treatment due to rapid drug degradation, poor targeting, and localized toxicity. This study explores a nanotechnology-based approach by developing and evaluating 5-FU loaded AgNP (AgNP–5FU) as a controlled drug delivery system. AgNP (AgNP) were synthesized and characterized using scanning electron microscopy (SEM), energy dispersive X-ray (EDX) analysis, particle size, and zeta potential analysis, while Fourier Transform Infrared Spectroscopy (FTIR) analysis suggested possible interaction between 5-FU and the …

Development And In Vitro Characterization Of 5-Fluorouracil–Loaded Plga Nanoparticles For Controlled Drug Delivery, Kavya Yandapalli

Theses, Dissertations and Capstones

Non-Alcoholic Fatty Liver Disease (NAFLD) is a progressive metabolic disorder characterized by excessive lipid accumulation in hepatocytes and chronic inflammation. Nanoparticle-based drug delivery systems offer a promising strategy to enhance therapeutic efficacy while minimizing systemic toxicity. The objective of this study is to formulate and characterize 5-FU–loaded PLGA nanoparticles and evaluate their in vitro performance. 5-FU–loaded PLGA nanoparticles were prepared using the double emulsion solvent evaporation method. The nanoparticles were characterized by particle size, polydispersity index, zeta potential and SEM. Drug encapsulation efficiency, loading capacity and in vitro drug release studies were conducted. The formulated nanoparticles exhibited sizes within the …

Sglt2 Inhibitors In Cardio-Oncology: A Systematic Review And Meta-Analysis, Luigi Spadafora, Marco Bernardi, Gianmarco Sarto, Beatrice Simeone, Erica Rocco, Salvatore Carbone, Laura Adelaide Dalla Vecchia, Roberto Franco Enrico Pedretti, Valentina Valentine, Rachele Di Mario, Maurizio Forte, Giacomo Frati, Antonio Abbate, Michele Golino, Francesco Versaci, Mariangela Peruzzi, Pierre Sabouret, Giuseppe Biondi Zoccai, Sebastiano Sciarretta

EVMS School of Health Professions Faculty Publications

Background

According to available evidence, sodium-glucose cotransporter-2 inhibitors (SGLT2i) may confer cardioprotection in patients with cancer undergoing chemotherapy.

Objectives

The objective of the study was to evaluate the impact of SGLT2i on all-cause mortality and heart failure (HF) outcomes in this population.

Methods

We searched PubMed, Cochrane CENTRAL, and Embase through August 2025 for studies of SGLT2i in adult patients with cancer. Random-effects models were used to pool effects for all-cause mortality and an HF composite (new-onset HF and/or HF hospitalization) in cancer patients >18 years. Meta-regression tested effect modification by beta-blockers, statins, angiotensin-converting enzyme inhibitors/angiotensin receptor blockers, age, and …

Tramadol For Treatment Of Neurogenic Cough: A Retrospective Case Series, Benjamin Joseph Rubinstein, Sheela Saidha, John Todd Sinacori

Department of Otolaryngology (ENT) Faculty Publications

Objectives

Explore the efficacy of tramadol for neurogenic cough and describe the longitudinal treatment experience.

Methods

A retrospective case series of adults with chronic cough who were treated with tramadol for neurogenic cough. A complete response was defined as no pathologic coughing, and a partial response was defined as 50% or greater reduction in severity or frequency.

Results

Sixty-nine patients were included. 38% of patients reported a complete response with an additional 33% reporting a partial response. The most common successful dosing regimen was 50 mg BID. Age, gender, and BMI did not impact treatment response. Patients with a history …

Circulating Biomarker Results From A Phase 2 Study Of Seralutinib In Pulmonary Arterial Hypertension, Robin Osterhout, Athénaïs Boucly, Raymond L. Benza, Richard N. Channick, Kelly M. Chin, Robert P. Frantz, Anna R. Hemnes, Luke S. Howard, Vallerie V. Mclaughlin, Olivier Sitbon, Jean-Luc Vachiéry, Rotham T. Zamanian, Richard Aranda, Matt Cravets, Zhaoqing Ding, Thao Duong-Verlé, David Mattola, Robert F. Roscigno, Ravikumar Sitapara, Lawrence S. Zisman, Jean-Marie Bruey, Hossein-Ardeschir Ghofrani

Department of Medicine Faculty Publications

[Introduction] To the Editor: Pulmonary arterial hypertension (PAH) is a progressive disease characterized by obstructive pulmonary arterial remodeling (1). Seralutinib, an investigational inhaled tyrosine kinase inhibitor, potently and selectively targets kinases relevant to PAH pathobiology including platelet-derived growth factor receptors (PDGFR) α and β, colony stimulating factor 1 receptor (CSF1R), and mast/stem cell growth factor receptor kit (c-KIT) (2). In preclinical models, seralutinib improved cardiopulmonary hemodynamics, reversed pulmonary vascular pathology and decreased right ventricular hypertrophy (3). In TORREY, a phase 2, multicenter, double-blind, randomized, placebo-controlled trial in PAH, seralutinib significantly reduced pulmonary vascular resistance (PVR) after 24 weeks with good …

Blister Packaging Versus Bottles: Caregiver Accuracy And Child Safety In Pediatric Medication Gummy Administration, Paolo Olimpiada

Nursing | Senior Theses

The safety of medication in pediatric settings has become an increasing concern as gummy-formulated medications have gained popularity in children’s care. Gummy-formulated medications have a close resemblance to candy, and this may mislead children to ingest additional doses or more than what was prescribed. Children also depend a lot on their caregivers for medications. Therefore, the design of packaging would be impactful in supporting medication dosing accuracy. And studies have evaluated safety features of packaging related to adherence, misuse, and unintended administration. However, limited studies have examined the influence of medication packaging on the dosing accuracy at the time of …