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New Drugs 2018, part 3.

This article discusses eight drugs recently approved by the FDA, including their indications and contraindications, precautions, dosage, and nursing considerations. The article also includes summary charts on 14 recently approved antineoplastic drugs and four drugs approved for rare disorders.

A cross-national review of novel oncology approvals in the United States and China (2020-2025).

INTRODUCTION: The United States and China are among the largest hubs for oncology drug development and commercialization. We conducted a comprehensive characterization of how the 2 largest oncology drug markets are evolving relative to one another, with implications for regulators, sponsors, clinicians, and patients. METHODS: Using publicly available drug databases from the US Food and Drug Administration (FDA) and the National Medical Products Administration (NMPA), we collected information on novel oncology approvals in the United States and China from 2020 to 2025. RESULTS: From 2020 to 2

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… Appendix Table AS1 lists all FDA and NMPA novel oncology drug approvals included in our analysis. … Drugs approved by both the FDA and the NMPA Between 2020 and 2025, 14 novel oncology drugs were approved by both the FDA and the NMPA ( Table 3 ). Drugs approved by either agency outside the study period were not included in this analysis. …

The prevalence and cost of unapproved uses of top-selling orphan drugs.

IntroductionThe Orphan Drug Act encourages drug development for rare conditions. However, some orphan drugs become top sellers for unclear reasons. We sought to evaluate the extent and cost of approved and unapproved uses of orphan drugs with the highest unit sales.MethodsWe assessed prescription patterns for four top-selling orphan drugs: lidocaine patch (Lidoderm) approved for post-herpetic neuralgia, modafinil (Provigil) approved for narcolepsy, cinacalcet (Sensipar) approved for hypercalcemia of parathyroid carcinoma, and imatinib (Gleevec) approved for chronic myelogenous leukemia and gas

Resmetirom: An Update on Therapy for Metabolic Dysfunction-Associated Steatohepatitis (MASH).

Metabolic dysfunction associated steatotic liver disease (MASLD) affects nearly one-third of adults worldwide, particularly among individuals with obesity or type 2 diabetes, and in 10-30% progresses to metabolic dysfunction associated steatohepatitis (MASH) or fibrosis. Resmetirom, a selective thyroid hormone receptor-β (THR-β) agonist, became the first therapy to demonstrate both MASH resolution and fibrosis improvement, leading to accelerated food and drug administration (FDA) approval in 2024 and conditional European Medicine Agency (EMA) approval in 2025 in the treatment of fibrotic MASH.

Is having more preapproval data the best way to assure drug safety?

An intensified focus on drug safety often leads to demands for more data collection prior to drug approval. Other approaches can be used, such as enhanced postmarketing surveillance. Many drug benefits and adverse effects are unlikely to become apparent before wide distribution among diverse patients. The best balance of pre- and postapproval data collection may vary by drug. The consequences of alternative strategies are complex and not always immediately apparent, so formal modeling offers the best approach to determine which strategy is optimal in each case.

Advancing cancer therapeutics: new perspectives on mechanisms, regulatory and policy challenges, and innovative multi-modality strategies for the early detection of cardiotoxicity.

Recent advances in cancer therapeutics, particularly targeted agents and tissue agnostic immunotherapies, have transformed care while introducing new challenges in cardiovascular safety. Although clinical guidelines have evolved, regulatory processes critical to ensuring safe innovation remain under-leveraged and often disconnected from real-world implementation. This gap is especially concerning amid federal downsizing, which has led to significant staff reductions at the United States Food and Drug Administration (FDA) and other health agencies. To meet growing needs in patient safety and th

Quantitative Medicine to Support Drug Development and Regulatory Decisions: A UK Regulatory Approach Informed by ICH M15.

Quantitative medicine supports modern drug development and regulatory decision-making by integrating non-clinical and clinical data to inform dose selection, trial optimization, and extrapolation across populations and benefit-risk assessment. The Medicines and Healthcare products Regulatory Agency (MHRA) has adopted quantitative approaches across the product lifecycle within a risk-proportionate regulatory framework. Internationally, the ICH M15 guideline provides a harmonized framework for planning, evaluation and documentation of model-informed drug development (MIDD) evidence which is alig

Properties of FDA-approved small molecule protein kinase inhibitors: A 2026 update.

Because of the dysregulation of protein kinase activity in many neoplastic and inflammatory diseases, protein kinases are among the most significant drug targets in the 21st century. Of the 94 FDA-approved protein kinase inhibitors, ten were approved in 2025. Of these drugs, six target dual specificity protein kinases (MEK1/2), fourteen inhibit protein-serine/threonine kinases, twenty-six block nonreceptor protein-tyrosine kinases, and 48 target receptor protein-tyrosine kinases. Most of these drugs (≈ 80) are prescribed for the management of neoplasms and others are used for the treatment of

The Impact on Acne Treatment Regimens if Benzoyl Peroxide-Containing Products are Removed From the Market.

Benzyl peroxide (BPO) has been an important component of many acne treatment regimens. However, an independent testing laboratory, Valisure, filed a Citizen’s Petition with the US Food and Drug Administration (FDA) requesting the removal of BPO products from the market. Their testing demonstrated that when exposed to elevated temperatures which could occur during shipping, marketed BPO products can form over 800 times the FDA concentration limits of benzene, a class 1 carcinogen. In this review, we aim to describe what a post-BPO removal acne treatment regimen might look like based on cu

Approved and Pipeline Pharmacological Interventions for Eating Disorders (2010-2025): 15 Years of Progress (or Lack Thereof).

Eating disorders (EDs) are complex psychiatric conditions characterized by disruptions in eating behaviors, body image concerns, and profound medical and psychosocial consequences. Despite their significant global prevalence, coupled with high morbidity and mortality rates, pharmacological treatment options remain limited. This review synthesizes evidence from clinical drug trials conducted between 1 January 2010 and 1 January 2025, supplemented with relevant literature, to evaluate the current and emerging pharmacological landscape for EDs. A systematic search of the U.S. Clinical Trials Regi

Iptacopan: First Approval.

Iptacopan (FABHALTA®) is an oral complement Factor B inhibitor developed by Novartis Pharmaceuticals for the treatment of complement-mediated diseases. Acting upstream of complement 5 in the alternative pathway, iptacopan inhibits both terminal complement-mediated intravascular haemolysis and complement 3-mediated extravascular haemolysis. On 5 December 2023, iptacopan received approval in the USA for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). This article summarizes the milestones in the development of iptacopan leading to this first approval for PNH.

Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses.

Mucopolysaccharidoses (MPS) disorders are a group of ultra-rare, inherited, lysosomal storage diseases caused by enzyme deficiencies that result in accumulation of glycosaminoglycans (GAGs) in cells throughout the body including the brain, typically leading to early death. Current treatments do not address the progressive cognitive impairment observed in patients with neuronopathic MPS disease. The rarity and clinical heterogeneity of these disorders as well as pre-existing brain disease in clinically diagnosed patients make the development of new therapeutics utilizing a traditional regulator

Properties of FDA-approved small molecule protein kinase inhibitors: A 2020 update.

Because genetic alterations including mutations, overexpression, translocations, and dysregulation of protein kinases are involved in the pathogenesis of many illnesses, this enzyme family is currently the subject of many drug discovery programs in the pharmaceutical industry. The US FDA approved four small molecule protein kinase antagonists in 2019; these include entrectinib, erdafitinib, pexidartinib, and fedratinib. Entrectinib binds to TRKA/B/C and ROS1 and is prescribed for the treatment of solid tumors with NTRK fusion proteins and for ROS1-postive non-small cell lung cancers. Erdafitin

A Drug to Prevent Pediatric Myopia-What Would it Take?

Because of the epidemic of myopia with both its short-term and long-term effects, we desperately need ways to slow myopic progression. In this study which was part of a myopia prevention symposium, the author answers the following question: Assuming that researchers did come up with a pharmacological treatment to slow myopic progression-what would it take to obtain regulatory approval from the United States Food and Drug Administration (FDA)? Previous publicly available information (a 2003 FDA advisory committee on this topic, International Conference on Harmonisation guidances on drug develop

Addressing a Yellow Fever Vaccine Shortage - United States, 2016-2017.

Recent manufacturing problems resulted in a shortage of the only U.S.-licensed yellow fever vaccine. This shortage is expected to lead to a complete depletion of yellow fever vaccine available for the immunization of U.S. travelers by mid-2017. CDC, the Food and Drug Administration (FDA), and Sanofi Pasteur are collaborating to ensure a continuous yellow fever vaccine supply in the United States. As part of this collaboration, Sanofi Pasteur submitted an expanded access investigational new drug (eIND) application to FDA in September 2016 to allow for the importation and use of an alternative y

One Health approach to Rift Valley fever vaccine development.

Since its discovery in the 1930s, Rift Valley fever virus (RVFV) spread across the African continent and invaded the Arabian Peninsula and several islands off the coast of Southeast Africa. The virus causes recurrent outbreaks in these regions, and its continued spread is of global concern. Next-generation veterinary vaccines of improved efficacy and safety are being developed that can soon be used for the widespread vaccination of livestock. However, due to regulatory and economic challenges, vaccine manufacturers have been reluctant to develop a human vaccine. Recent innovations in veterinar