FDA Extends Review Period for Gene Therapies for Beta-Thalassemia, Cerebral Adrenoleukodystrophy
The FDA extended the target dates for beti-cel and eli-cel to allow time to review additional clinical information for both applications.
Bleeding Disorders
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.
Fitusiran Prophylaxis Improves Annualized Bleeding Rate in Hemophilia A and B
Researchers sought to determine whether fitusiran prophylaxis would reduce the rate of bleeding events among patients with hemophilia A or B with inhibitors.
Novel NMDA Receptor Blocker Designated Orphan Drug for Subarachnoid Hemorrhage
NP10679 is an investigational subunit-specific NMDA receptor inhibitor.
One-Time Gene Therapy Gets Priority Review for Beta-Thalassemia
Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells.
FDA Clears System That Allows for Remote Endoscopy Procedures
PillCam SB3 @HOME offers a telehealth option for direct visualization and monitoring of the small intestine.
GI Bleeding Up for New Users of Rivaroxaban
Compared with other DOACs, rivaroxaban linked to higher rates of GI bleeding overall and in an analysis restricted to patients with atrial fibrillation
Cinacalcet Does Not Up Risk for Severe Gastrointestinal Bleeding in Hemodialysis
Study is the largest to explore this potential safety issue to date.
FDA Issues Safety Alert Regarding Use of Hydroxyethyl Starch Products
There are currently 4 FDA-approved hydroxyethyl starch products on the market.
Vonvendi Under Review for Prophylactic Use in von Willebrand Disease
The sBLA submission is supported by data from a phase 3 study that assessed the efficacy and safety of prophylactic treatment with Vonvendi in adults with severe VWD over a period of 12 months.
