Hemlibra Clinical Development Timeline
Chronicle of the development and US FDA review of Genentech's emicizumab-kxwh for hemophilia A.
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The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.
The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.
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While the first six months of the year saw eight new drugs targeting a range of diseases enter the European Medicines Agency’s priority medicines scheme, and three PRIME-designated treatments go on to win EU marketing approval, use of the accelerated assessment mechanism appears to be limited.
The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.
The Pediatric Advisory Committee supported routine safety monitoring for three vaccines and 21 drugs, but the panel’s consumer representative said adverse event reporting needs improvement and FDA literature reviews should include lawsuits as well as social media.