Xembify Approved to Treat Primary Humoral Immunodeficiency
The approval was based on a prospective, open label, single arm, multicenter clinical trial that evaluated the safety and efficacy of Xembify compared with Gamunex-C.
Immune Disorders
First Treatment for Neuromyelitis Optica Spectrum Disorder Approved
The FDA has approved Soliris (eculizumab; Alexion Pharmaceuticals, Inc) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
Rituxan Gets Priority Review for Pediatric Granulomatosis With Polyangiitis and Microscopic Polyangiitis
The FDA has accepted for Priority Review the supplemental BLA for Rituxan for use in combination with glucocorticoids for the treatment of granulomatosis with polyangiitis and microscopic polyangiitis in children ≥2 years old.
FDA Accepts BLA for Tissue Based Regenerative Tx for Pediatric Congenital Athymia
The FDA has accepted for Priority Review the Biologics License Application for RVT-802 (Enzyvant), an investigational tissue-based regenerative therapy for pediatric congenital athymia.
Jakafi Approved for Steroid-Refractory Acute Graft-Versus-Host Disease
The FDA has approved Jakafi (ruxolitinib; Incyte) for treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients ≥12 years old.
Ruzurgi Approved for Pediatric Patients With Lambert-Eaton Myasthenic Syndrome
The FDA has approved Ruzurgi (amifampridine; Jacobus Pharmaceutical) for the treatment of Lambert-Eaton myasthenic syndrome in patients 6 to <17 years of age.
Novel Treatment for Neuromyelitis Optica Spectrum Disorder Gets Breakthrough Status From FDA
The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to inebilizumab (Viela Bio), an investigational monotherapy for neuromyelitis optica spectrum disorder (NMOSD).
Elafibranor Gets Breakthrough Therapy Designation for Primary Biliary Cholangitis
The FDA has granted Breakthrough Therapy designation to elafibranor for the treatment of primary biliary cholangitis (PBC) in adults with inadequate response to ursodeoxycholic acid (UDCA).
Is There Evidence to Support Kombucha Health Claims in Humans?
According to the results of a systematic review, there is a lack of evidence supporting the potential health benefits of kombucha, a fermented tea beverage, in humans.
Asceniv Approved for Primary Humoral Immunodeficiency in Adolescents, Adults
ADMA Biologics announced that the FDA has approved Asceniv (immune globulin intravenous [human] – slra) 10% Liquid for the treatment of primary humoral immunodeficiency disease (PIDD) in patients aged ≥12 years.
