Gene Therapy Gets Fast Track Status for Hemophilia A
In preclinical studies, ASC618 has been shown to have at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered constructs.
Diagnosis & Disease Information
Once-Weekly Efanesoctocog Alfa Prevents Bleeding Episodes in Hemophilia A Trial
The phase 3 XTEND-1 study evaluated efanesoctocog alfa in 159 patients with severe hemophilia A who were previously treated with factor VIII replacement therapy.
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.
BeneFIX Label Updated to Remove Routine Prophylaxis in Children With Hemophilia B
The Agency stated that it “erred in approving the indication for routine prophylaxis in children with hemophilia B” due to the Orphan Drug exclusivity.
New Drugs Approved in 2020
Visit our New Drug Products page to stay up-to-date on the latest drug approvals.
FDA: Risk of Inadvertent Intrathecal Administration of Tranexamic Acid Injection
The FDA has received reports of tranexamic acid injection being erroneously administered intrathecally in the perioperative setting instead of the intended intrathecal anesthetic (eg, bupivacaine injection) for neuraxial anesthesia.
Immunogenicity Data Added to Nuwiq Labeling
The prospective, multinational, open-label, non-controlled phase 3 NuProtect study assessed the immunogenicity, efficacy and safety of Nuwiq in 105 previously untreated patients with severe hemophilia A.
MPR Weekly Dose Podcast Episode 48
Treatment shows promise for patients who had pneumonia associated with COVID-19; A COVID-19 test that may have given inaccurate results; Neck gaiters as face masks may actually spread the virus; Drug applications are halted by the FDA; And Narcan has it’s shelf life extended. Also, we recap drug pipeline news from the past week.
Xyntha Approved for Routine Prophylaxis in Hemophilia A
Xyntha works by temporarily replacing the missing clotting factor VIII that is needed for effective hemostasis.
