Although a decrease in sweat chloride levels was suggestive of benefit, this did not correlate with improvements in FEV1 function, FDA said. The agency also noted that the patient-reported outcomes instrument used in the pivotal clinical trials, though not fully validated, measured respiratory symptoms that are clinically relevant to patients.
Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Kalydeco (ivacaftor) demonstrated generally positive effects on a slew of secondary endpoints in two pivotal trials, and yet it remains a question mark how, and whether, some of these measures translate into clinical benefit.
During the course of their review, FDA staff raised questions about the clinical meaningfulness of changes in sweat chloride and...
The Trump Administration’s rhetoric and policy approaches illustrate the confusion that often arises between efforts to cut drug prices and cutting spending.
Companies with significant exposure to Medicare and Medicaid have fewer options for passing along higher costs to consumers because of price controls in the programs.
The news coincides with rumors that Commissioner Martin Makary soon will name a new Center for Drug Evaluation and Research director.
US FDA critics have long decried the practice of using informal communications to convey regulatory expectations as podium policy. In the Makary era, the phrase may need to be updated because the agency now seems to be specializing in policy by podcast.
Tala Fakhouri said the agency’s use of the AI tool in the review process has generated lots of questions.
Kumar takes over as acting director of the Center for Biologics Evaluation and Research’s Office of Therapeutic Products days after director Nicole Verdun was ousted.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
