Exclusive: FDA staff struggle to meet product review deadlines after DOGE layoffs
By Patrick Wingrove
The Department of Government Efficiency, led by Elon Musk, fired around a thousand FDA employees in February 2025, leading the agency to spend longer on reviews. Some scientists have been assigned double the number of new product applications for review, and were instructed to shelve other work. This could result to products coming to market later, and increased risks of missing red-flags.
FDA approves drug for neurofibromatosis
By Max Barnhart
Mirdametinib, a drug once shelved by Pfizer, is now being repositioned and has been approved by the FDA for the treatment of neurofibromatosis type 1 (NF1) in adults and children. NF1 is a rare genetic condition in which tumours grow on Schwann cells, forming tumours around nerves. The drug, now developed by SpringWorks Therapeutics, results in patient’s tumour shrinking by 50-60%.
A New Pan-Coronovirus Protease Inhibitor Possibility
By Derek Lowe
The AI/computational group of the Drugs for Neglected Diseases Institute has been working on coronavirus protease inhibitors, aiming at making compounds active on more than just the SARS-CoV-2 virus. Derek Lowe shares his thoughts and questions about this new compound, which is a follow-up to a previous molecule that had significant PK issues and activation of PXR receptor.
A guide to kinase inhibitors in biotech: From cancer breakthroughs to autoimmune innovations
By Jules Adam
Kinases regulate many aspects of cellular function and therefore became widely targeted enzymes in drug development. This article reviews the broad research on kinases inhibitors in biotech, highlighting breakthrough therapies and exciting innovations from cancer therapies to autoimmune diseases treatment.
Immune Checkpoint Inhibitors and Their Side Effects
By The American Cancer Society
This article reviews several immune checkpoint inhibitors, also known as monoclonal antibodies, and their side effect. Monoclonal antibodies are used as cancer therapy to help the immune system to better find and destroy cancer cells, rather than kill them directly. However, those checkpoint inhibitors can have side effects similar to an allergic reaction, or an autoimmune reaction.
M15 General Principles for Model-Informed Drug Development
By The FDA
This guideline provides general recommendations for planning, model evaluation, and documentation of evidence derived from Model-Informed Drug Development (MIDD), hereafter “MIDD evidence.” It establishes a harmonized assessment framework (including associated terminology) for MIDD evidence.
Draft guidance on individualised mRNA cancer immunotherapies
By Medicines and Healthcare products Regulatory Agency
Individualised mRNA cancer immunotherapies (colloquially referred to as personalised cancer vaccines) are a new type of cancer treatment that use mRNA technology. Because each patient receives a slightly different version of the mRNA therapy, matching their tumour fingerprint, there is an urgent need for guidance on the unique regulatory challenges of this approach. This draft guideline outlines a clear and streamlined regulatory pathway to approval.
Points to consider for revising the ICH S7A guideline on safety and secondary pharmacology
By Jean-Pierre Valentin and Derek Leishman
The authors of this paper propose a revision of the ICH S7A guideline on safety pharmacology, to address its limitations and further reduce adverse drug reactions and attritions. The revised guideline should aim to address key aspects of safety and secondary pharmacology. Further considerations are suggested to help optimize drug safety evaluations and align industry practices with modern scientific advancements and ethical considerations.
Painkiller mimics cannabis without the psychoactive side effects
By Bethany Halford
A molecule that targets the same receptor as the active ingredient in cannabis could lead scientists to new drugs for chronic pain. Chemists designed the compound, known as VIP36, to bind cannabinoid receptor 1 (CB1) in the body but not in the brain. This design prevents the molecule from having psychoactive side effects. In mouse studies, VIP36 treated inflammatory pain, nerve pain, and migraines.