Biomarkers for Precision Patient Selection in Cancer Therapy Approvals in the US, from 2011 to 2023.

During the period of 2011-2023, the US Food and Drug Administration (US FDA) granted 139 accelerated and 329 regular approvals for 86 and 152 cancer therapeutic products, respectively. The percentage of approvals for a biomarker-defined population was numerically higher in accelerated approvals in comparison to regular approvals, that is, 48% vs. 40%.

Pharmacogenetic Labeling of FDA-Approved Drugs: A Regulatory Retrospective.

The U.S. Food and Drug Administration recently marked 10 years since first updating the labeling for warfarin (often referred to as the "poster child" of pharmacogenomics) to include information regarding the potential impact of and genetic variation on warfarin dosing requirements and risks.

Pharmacogenetics in the evaluation of new drugs: a multiregional regulatory perspective.

Pharmacogenetics, one of the cornerstones of personalized medicine, has the potential to change the way in which health care is offered by stratifying patients into various pretreatment categories, such as likely responders, likely non-responders or likely to experience adverse drug reactions. In order to advance drug development and regulatory science, regulatory agencies globally have promulgated guidelines on pharmacogenetics for nearly a decade. The aim of this article is to provide an overview of new guidelines for the implementation of pharmacogenetics in drug development from a multiregional regulatory perspective - encompassing Europe, the United States and Japan - with an emphasis on clinical pharmacokinetics.

Individualizing therapy of monoclonal antibodies and fusion proteins: emerging potential in the age of personalized medicine.

Personalized medicine is an emerging and promising alternative to standard therapy regimens with the potential to significantly influence therapeutic interventions for many diseases. An extensive literature review of studies that focused on pharmacogenomics of monoclonal antibodies (mAbs) and immunoglobulin-containing fusion proteins (igFPs) was conducted. A comprehensive survey of the US FDA-approved labels revealed that pharmacogenomics information has also been incorporated into the label of some mAbs to guide therapy.

Alpha-1 antitrypsin protein and gene therapies decrease autoimmunity and delay arthritis development in mouse model.

Background: Alpha-1 antitrypsin (AAT) is a multi-functional protein that has anti-inflammatory and tissue protective properties. We previously reported that human AAT (hAAT) gene therapy prevented autoimmune diabetes in non-obese diabetic (NOD) mice and suppressed arthritis development in combination with doxycycline in mice. In the present study we investigated the feasibility of hAAT monotherapy for the treatment of chronic arthritis in collagen-induced arthritis (CIA), a mouse model of rheumatoid arthritis (RA).

Combination of alpha-1 antitrypsin and doxycycline suppresses collagen-induced arthritis.

Background: Rheumatoid arthritis (RA) is a complex disease characterized by autoimmune inflammation and joint destruction. Despite recent advances in RA treatment, current therapies require further improvement to overcome adverse events and ineffectiveness in some cases. By targeting different pathways/molecules using drug combinations, a better treatment can be obtained, whereas adverse events are reduced.