Review
Clinical Neurology
Leigh K. Sharpless, Aaron S. Kesselheim, Serena L. Orr, Jonathan Darrow
Summary: This study assessed the characteristics and variation of endpoints used in trials supporting FDA approval of medications for migraine treatment. The findings revealed inconsistent endpoint selection within the same indication, mechanistic class, and route of administration, making it difficult to directly compare these medications. Inconsistent definitions for episodic and chronic migraine were also observed.
Editorial Material
Clinical Neurology
Juliana H. Vanderpluym
Summary: Migraine management includes acute treatments for immediate relief and preventive treatments for long-term reduction of attacks. Individualized treatment decisions require consideration of factors such as efficacy, safety, characteristics, and patient preferences.
Article
Pharmacology & Pharmacy
Hideki Maeda, Yuka Fukuda, Marika Uchida
Summary: The public knowledge-based application in Japan aims to eliminate off-label use due to drug lag. A comprehensive investigation over the past two decades found that more anticancer and pediatric drugs were approved through this system, with shorter review times compared to normal applications. Additionally, only one drug for orphan diseases was approved through this system.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2021)
Review
Oncology
Aleksandra Sochacka-Cwikla, Marcin Maczynski, Andrzej Regiec
Summary: This article summarizes the new drugs used in the treatment of hematological malignancies in the past decade, including small molecule and macromolecule agents. The development of these drugs has improved the clinical outcomes for blood cancers.
Review
Pharmacology & Pharmacy
Davide Benedetto Tiz, Luana Bagnoli, Ornelio Rosati, Francesca Marini, Claudio Santi, Luca Sancineto
Summary: This review describes recently FDA-approved drugs, many of which contain active moieties that have not been previously approved by the FDA. These drugs provide important new therapies for patients with multiple unmet diseases.
Letter
Medicine, General & Internal
S. Sean Tu, Aaron S. Kesselheim, Kathrine Wetherbee, William B. Feldman
Summary: This study evaluates the frequency of continuation patents on brand-name drugs approved by the US Food and Drug Administration from 2000 to 2015.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2023)
Article
Oncology
Marjorie E. E. Zettler
Summary: This study aims to determine the tumor types represented and method of mutation identification in trials supporting FDA approvals of tissue-agnostic drugs, as well as the post-marketing requirements and commitments issued for additional studies and validation of companion diagnostic devices. It was found that between 2017 and 2022, six drugs were approved for seven tissue-agnostic indications, with 9-15 different tumor types represented in the supporting trials. Most drug approvals did not have concurrent approval of a diagnostic test.
Article
Health Care Sciences & Services
Victor L. Van de Wiele, Andrew W. Torrance, Aaron S. Kesselheim
Summary: Newly approved drugs are mostly protected by patents, which mainly cover chemical compounds and methods of use. The median time for patents to expire after the approval of original drugs is 10.3 years. About one in five patents have no counterparts in other major jurisdictions, suggesting different manufacturer strategies or patent standards in the US compared to other comparable settings. Patent protection for newly approved drugs largely focuses on product and its uses, indicating that patent reform is crucial for timely entry of generic drugs.
Review
Chemistry, Medicinal
Sheng Han, Yiming Lu
Summary: Human immunodeficiency virus (HIV) is the cause of acquired immunodeficiency syndrome (AIDS). The FDA has approved over thirty antiretroviral drugs, one-third of which contain fluorine atoms. The addition of fluorine to drug compounds is a well-known strategy in medicinal chemistry. This review summarizes 11 fluorine-containing anti-HIV drugs, focusing on their efficacy, resistance, safety, and the role of fluorine in their development. These examples may aid in the discovery of new drug candidates with fluorine-containing structures.
EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY
(2023)
Review
Pharmacology & Pharmacy
Cathy Anne Pinto, Zaneta Balantac, Shahrul Mt-Isa, Xinyue Liu, Oswaldo L. Bracco, Harrison Clarke, Tommi Tervonen
Summary: The policies of EMA and FDA aim to strengthen benefit-risk capabilities, but how they are implemented in regulatory practice is unclear. A systematic review of oncology drug approvals between 2015 and 2020 revealed that the standard of evidence for benefit-risk assessments has diversified over time, but patient experience and real-world data are rarely included in these assessments despite policy goals to increase their use.
DRUG DISCOVERY TODAY
(2023)
Review
Pharmacology & Pharmacy
Chandani T. Muleva, Sonali S. Bharate
Summary: Salification is an important method to modulate the properties of poorly water-soluble drugs, and halogen acids play a significant role in the process. Analyzing data from the orange book, it was found that -600 pharmaceutical salts of new chemical entities have been approved in the past nine decades, with halides being the most common counterion. Among the halides, salts prepared with hydrochloric acid are the most dominant. Despite their corrosive and hazardous nature, halogen acids are necessary in salt screening experiments for drug discovery and development.
JOURNAL OF DRUG DELIVERY SCIENCE AND TECHNOLOGY
(2023)
Article
Chemistry, Multidisciplinary
Jingrui He, Ziyi Li, Gagan Dhawan, Wei Zhang, Alexander E. Sorochinsky, Greg Butler, Vadim A. Soloshonok, Jianlin Han
Summary: This review article presents nine newly approved fluorine-containing drugs by the US Food and Drug Administration (FDA) in 2021. These small molecule drugs feature aromatic fluo-rine, trifluoromethyl, and chlorodifluoro groups. The therapeutic areas of these fluorine-containing drugs include multiple myeloma, lymphoma, HIV, chronic heart failure, chronic myeloid leukemia, (ANCA)-associated vasculitis, migraines, von Hippel-Lindau disease, and non-small cell lung cancer. The review also discusses the brief biological activities and synthetic methods for each of these nine drugs.
CHINESE CHEMICAL LETTERS
(2023)
Article
Oncology
Yifei Xie, Ke Yao, Zigang Dong, Kangdong Liu
Summary: Proliferating cancer cells have metabolic alterations and specific nutritional needs, making targeting metabolic abnormalities for cancer chemoprevention promising. Many FDA-approved anti-metabolic drugs have been shown to decrease tumor risk, inhibit tumor growth, or enhance the effect of chemotherapy drugs, with fewer side effects compared to chemotherapeutic drugs.
Review
Medicine, Research & Experimental
Ademola C. Famurewa, Anirban Goutam Mukherjee, Uddesh Ramesh Wanjari, Aarthi Sukumar, Reshma Murali, Kaviyarasi Renu, Balachandar Vellingiri, Abhijit Dey, Abilash Valsala Gopalakrishnan
Summary: Platinum-based anticancer drugs have been widely used for treating various cancer types, but their toxicity to non-targeted organs is a concern. Research has shown that this toxicity is associated with free radical generation, DNA impairment, mitochondrial dysfunctions, and other factors. Repurposing non-anticancer drugs has emerged as a strategy to mitigate the side effects of platinum-based drugs.
Review
Engineering, Chemical
Aathira Sujathan Nair, Ashutosh Kumar Singh, Astik Kumar, Sunil Kumar, Sunitha Sukumaran, Vishal Payyalot Koyiparambath, Leena K. Pappachen, T. M. Rangarajan, Hoon Kim, Bijo Mathew
Summary: As people around the world consider 2020 as the year dominated by COVID-19, the medical community acknowledges it as one of the most successful years for drug approvals by the FDA, along with 1996. Both years witnessed the approval of 53 new drug molecules. In 2020, 53 new chemical entities and 13 biological medicines were approved, with a significant number of them containing fluorine or fluorine-containing functional groups and exhibiting diverse pharmacological activities.
