The U.S. Food and Drug Administration announced another major step in its initiative to streamline the development of biosimilar medicines, which are like “generic” versions of biologic drugs. In new draft guidance issued today, the agency recommended streamlining unnecessary clinical pharmacokinetic (PK) testing when scientifically justified. This change could save...
“Today’s accelerated approval provides a breakthrough treatment for pediatric patients with severe Leukocyte Adhesion Deficiency Type I—the first FDA-approved gene therapy to treat this disease,” said Vinay Prasad, M.D., M.P.H., Chief Medical and Scientific Officer and Director of the FDA’s Center for Biologics Evaluation and Research. “The FDA continues to exercise significant regulatory flexibilities as applicable, during both Chemistry, Manufacturing and Control (CMC) and Clinical review of licensing applications. For rare diseases, the FDA considers small patient populations in clinical trials and all available sources of evidence to advance life-changing treatments while still meeting its rigorous scientific standards.”
Endometrial cancer is often treatable in early stages. But once it spreads — especially after chemotherapy and immunotherapy have already been used — treatment options become very limited. A new phase II study looked at whether a drug called sacituzumab govitecan, or SG, could help patients with advanced endometrial cancer who had already tried standard therapies. This research was part of the larger TROPiCS-03 clinical trial, which studied SG in different types of metastatic solid tumors.
Cannabis and Cannabis-derived products have become increasingly available in recent years, with new and different types of products appearing all the time. These products raise questions and concerns for many consumers. And if you are pregnant or breastfeeding, you might have even more questions about whether these products are safe for you. FDA strongly advises...
For years, we’ve been told that lowering “bad” cholesterol is the key to preventing heart attacks. But what if inflammation is an even bigger threat, especially if you’re already taking a statin?
A large analysis published in The Lancet looked at this exact question. Researchers studied 31,245 patients from three major clinical trials, PROMINENT, REDUCE-IT, and STRENGTH, all of whom were already taking statins to lower cholesterol.
The U.S. Food and Drug Administration issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations. The draft guidance, issued by the Center for Biologics Evaluation and Research and Center...