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.
Diagnosis & Disease Information
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.
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.
Neulasta Ad Claims Misleading, May Undermine Biosimilar Use, Says FDA
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.
Ayvakit Approved for Advanced Systemic Mastocytosis
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.
Ultomiris Approved for Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
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 Label Updated With Benefit Statement, Instructions for Use
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.
Nipocalimab Gets Fast-Tracked for Hemolytic Disease of the Fetus and Newborn
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).
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