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Following through on a 2023 pledge from its CEO, Amylyx is voluntarily pulling its controversial amyotrophic lateral sclerosis drug Relyvrio/Albrioza (AMX0035; ursodoxicoltaurine) after the drug failed its confirmatory phase 3 study. Read More
The FDA’s guidance on the quantity of bioavailability (BA) and bioequivalence (BE) testing samples to be retained by NDA applicants and contract research organizations (CRO) contains both draft and final language focusing on both the test article and reference standard. Read More
Closing on May 31, the European Medicines Agency (EMA) has opened a second consultation for its guideline on requirements for investigational advanced therapy medicinal products (ATMP) in clinical trials. Read More
In a nod to the increasing potential of demonstrating drug/biologic safety and/or effectiveness through noninterventional studies, the FDA has published long-awaited draft guidance on designing and analyzing such studies with real-world data (RWD) in mind. Read More
Last month, signs that magrolimab increases the risk of death prompted Gilead to shelve the drug for blood cancer, Vertex’s second-gen cystic fibrosis combo leaves the company’s first-gen efforts in the dust, a new drug combo looks promising for multiple myeloma, and Regenxbio hopes to snag an Accelerated Approval for a new rare disease gene therapy. Also, Astra Zeneca is touting “major advance” in treating one type of non-small cell lung cancer. Read More
AI models developed by GE HealthCare used readily available clinical data to forecast cancer immunotherapy outcomes and adverse reactions with 70-80 percent accuracy, according to an article published in the Journal of Clinical Oncology Clinical Cancer Informatics. Read More
The FDA has published new draft guidance on informed consent that lines up with revisions to the Common Rule made in 2017, offering up-to-date recommendations on starting the process with the sharing of essential clinical trial information in ways that patients can understand. Read More
A student group that filed a citizen petition aimed at improving what it alleges are “hugely deficient” trial reporting enforcement efforts by the FDA has gained some ground in the ongoing effort to increase trial transparency, with the agency granting one of its requests and pledging to consider another. Read More
Enhanced access to real world data (RWD) sources critical to regulators, pharma companies and researchers is the objective for the two electronic catalogues launched last week by the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA). Read More
The FDA’s draft guidance on its Advanced Manufacturing Technologies (AMT) pathway would be restrictive to manufacturers in the cell and gene therapy spaces, the Alliance for Regenerative Medicine and the American Society of Gene and Cell Therapy said in filed comments. Read More