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.
The sNDA and NDA are supported by data from the open-label phase 2a HOPE-KIDS1 study.
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 is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes.
The FDA noted that the application could not be approved in its present form and had requested an additional clinical study be conducted.
Eflapegrastim is a novel, long-acting G-CSF comprised of 2 protein components, an analogue of G-CSF and an Fc antibody fragment.
The ad claims that there is a higher risk of febrile neutropenia when pegfilgrastim is administered via prefilled syringe vs Neulasta Onpro on-body injector.
The approval was based on data from 2 multicenter, single-arm, open-label clinical trials that evaluated Ayvakit in 53 patients with advanced systemic mastocytosis.
The approval was based on data from an open-label, phase 3 study that assessed Ultomiris in 13 patients 9 through 17 years of age with PNH.
Endari (L-glutamine; Emmaus Medical) is an amino acid indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients aged 5 years and older.
The Food and Drug Administration (FDA) has granted Fast Track designation to nipocalimab (M281) for the prevention of hemolytic disease of the fetus and newborn (HDFN).