Prophylactic Steroids May Prevent Complications of Thyroidectomy
Rates of postoperative hypocalcemia and voice dysfunction lower with single preoperative dose of dexamethasone versus placebo
Hematologic Disorders
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.
Mitapivat Granted Priority Review for Pyruvate Kinase Deficiency
Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes.
MPR Weekly Dose Podcast Episode 89
Big news on the COVID-19 vaccine booster dose. The CDC issues new recommendations on vaccines. What protection the Moderna vaccine offers after 6 months. A treatment approved for Pompe disease. And the FDA denies an application for a new roxadustat indication.
FDA Denies Approval of Roxadustat for Anemia in CKD
The FDA noted that the application could not be approved in its present form and had requested an additional clinical study be conducted.
Manufacturing Deficiencies Delay Decision on Eflapegrastim
Eflapegrastim is a novel, long-acting G-CSF comprised of 2 protein components, an analogue of G-CSF and an Fc antibody fragment.
Midodrine Cuts Recurrent Syncope in Young, Healthy Patients
Absolute risk reduced by 19 percentage points, with number needed to treat to prevent syncope of 5.3
FDA Approves Berinert Administration Kit for Hereditary Angioedema
The FDA has approved the supplemental request for the co-packaging of a convenience administration kit for Berinert (C1 esterase inhibitor, [Human]) for acute abdominal, facial, or laryngeal attacks of hereditary angioedema.
Rezurock Approved for Chronic Graft-vs-Host Disease
The approval was based on data from a phase 2 trial that evaluated Rezurock in 65 patients with chronic GVHD who received 2 to 5 prior lines of systemic therapy.
