Rare Pediatric Disease Designation Archives

SpringWorks Therapeutics Initiates Rolling Submission of New Drug Application to the FDA for Mirdametinib for the Treatment of Children and Adults with NF1-PN

March 4, 2024March 4, 2024

SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the…

United States Food & Drug Administration (FDA) Grants Mesoblast Orphan-Drug Designation for Revascor® (Rexlemestrocel-L) in Children With Congenital Heart Disease

February 15, 2024February 15, 2024

Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food…

United States Food & Drug Administration (FDA) Grants Mesoblast Rare Pediatric Disease Designation for Revascor® (Rexlemestrocel-L) in Children With Congenital Heart Disease

January 19, 2024January 19, 2024

Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food…