Hemlibra Reviewers
Executive Summary
US FDA staff who participated in the review of Genentech's hemophilia A treatment emicizumab-kxwh.
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Genentech's Hemlibra: Clinical Outcome Assessment Data Only Partially Swayed US FDA
Hemophilia A drug's labeling reflects data on physical function improvement because FDA deemed that portion of the Haem-A-QoL instrument fit for purpose, while other questions were viewed as insensitive to change or irrelevant. Review documents suggest agency was unimpressed with results from a health status instrument frequently used in economic analyses.
US FDA Sought Patient Viewpoint On Hemlibra's Benefit/Risk Profile
Hemophilia patient advocate expressed concerns to FDA about ability of hemophilia treatment centers to educate patients about thrombotic microangiopathy and thromboembolic events, unexpected but serious cases of which were seen in the clinical program.
First Three Novel Biologic Suffixes Came Courtesy Of US FDA, Not Sponsors
FDA assigned a four-letter nonproprietary name suffix to Genentech's Hemlibra because an expedited review left little time to evaluate the sponsor's late-submitted proposals; UltraGenyx asked FDA to assign one for Mepsevii, while Spark submitted 10 suffix candidates for Luxturna only to see all of them rejected for being too meaningful.