World’s First Gene Therapies Approved to Treat Sickle Cell Disease
The U.S. Food and Drug Administration has approved two treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years and older.
FDA Approves Repotrectinib for Non-Small Cell Lung Cancer
The U.S. Food and Drug Administration has approved Bristol Myers Squibb’s repotrectinib (Augtryo) for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer.
Fresenius Kabi’s Tyenne, a Biosimilar of Actemra, Is Approved to Treat Autoimmune Diseases
The U.S. Food and Drug Administration has approved Tyenne (tocilizumab-aazg), a biosimilar referencing tocilizumab (Actemra; Genentech), to treat multiple autoimmune diseases, including rheumatoid arthritis and juvenile idiopathic arthritis.
Hizentra Vials to Be Discontinued and Replaced with Prefilled Syringes
CSL Behring is discontinuing all sizes of Hizentra vials in the U.S. by the end of September 2024.
Selarsdi Approved as Biosimilar to Stelara to Treat Plaque Psoriasis and Psoriatic Arthritis
The U.S. Food and Drug Admini-stration has approved Selarsdi (ustekinumab-aekn) injection for subcutaneous use, as a biosimilar to Stelara, for the treatment of moderate to severe plaque psoriasis and for active psoriatic arthritis in adults and pediatric patients 6 years and older.
Blood Test Predicts Multiple Sclerosis Years Before Symptoms Appear
Researchers at the University of California, San Francisco, have identified a specific pattern of autoantibodies in the blood that precedes the clinical onset of multiple sclerosis.
FDA Grants wilate Orphan Drug Exclusivity
The U.S. Food and Drug Administration (FDA) has granted orphan drug exclusivity for Octapharma’s wilate, von Willebrand factor/coagulation factor VIII complex (human) lyophilized powder for solution for intravenous injection, for routine prophylaxis to reduce the frequency of bleeding episodes in adults and children 6 years of age and older with von Willebrand disease (VWD).
New Rule Expands Access to Health Information and Improves Prior Authorization Process
CMS finalized the CMS Interoperability and Prior Authorization Final Rule to improve the electronic exchange of health information and prior authorization processes for medical items and services.
Albumin Administration Superior to Ringer’s Lactate for Maintaining Normovolemia During Major Surgical Hemorrhage
Investigators at the Bern University Hospital conducted a single-center randomized clinical trial to quantify and compare the PVE properties of iso-oncotic 5% albumin, hyper-oncotic 20% albumin and Ringer’s lactate (RL) in patients undergoing radical cystectomy.
Emicizumab Prophylaxis Efficacious and Well-Tolerated in Infants with Severe Hemophilia A Without Inhibitors
The Phase IIIb study of emicizumab shows promise for reducing risk of spontaneous and traumatic bleeds in infants with hemophilia A.
