Zydelig US Indications Withdrawn for Follicular Lymphoma, Small Lymphocytic Leukemia
Continued approval required further verification and description of clinical benefit in confirmatory trials.
Continued approval required further verification and description of clinical benefit in confirmatory trials.
The phase 3 GENEr8-1 study evaluated valoctocogene roxaparvovec in adults with severe hemophilia and residual FVIII levels less than or equal to 1 IU/dL.
Injectafer is an iron replacement product.
The approval was based on data from the European Sickle Cell Disease Cohort study, which evaluated the efficacy and safety of Siklos in 1077 adults with sickle cell disease.
Exposure to higher temperatures may have significantly impacted the efficacy of the recalled product.
The application is supported by data from the phase 2 BEYOND study.
Narsoplimab is a human monoclonal antibody that specifically targets mannan-binding lectin-associated serine protease-2.
The approval was based on data from the phase 3 REACH3 study which included 329 patients.
The sNDA and NDA are supported by data from the open-label phase 2a HOPE-KIDS1 study.
The supplemental Biologics License Application is supported by data from the phase 2 ABA2 trial (ClinicalTrials.gov Identifier: NCT01743131) and real world evidence from a registry trial.