Zydelig US Indications Withdrawn for Follicular Lymphoma, Small Lymphocytic Leukemia
Continued approval required further verification and description of clinical benefit in confirmatory trials.
Diagnosis & Disease Information
Durable Response Observed With Gene Therapy for Hemophilia A
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 Approval Expanded to Pediatric Patients With Iron Deficiency Anemia
Injectafer is an iron replacement product.
Siklos Approval Expanded to Include Adults With Sickle Cell Anemia
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.
High Temperature Exposure Prompts Recall of Anticoagulant Med
Exposure to higher temperatures may have significantly impacted the efficacy of the recalled product.
Luspatercept Under Review for Nontransfusion Dependent Beta Thalassemia
The application is supported by data from the phase 2 BEYOND study.
FDA Unable to Approve Narsoplimab for HSCT-TMA Without More Data
Narsoplimab is a human monoclonal antibody that specifically targets mannan-binding lectin-associated serine protease-2.
Jakafi Approved for Chronic Graft-vs-Host Disease
The approval was based on data from the phase 3 REACH3 study which included 329 patients.
Voxelotor Gets Priority Review for Pediatric Sickle Cell Disease
The sNDA and NDA are supported by data from the open-label phase 2a HOPE-KIDS1 study.
Abatacept Gets Priority Review for Moderate to Severe Acute Graft vs Host Disease
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.
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