United States Food and Drug Administration
FDA Releases Second Batch of Complete Response Letters, Moves to Real-Time Public Disclosure
FDA; Complete Response Letters; CRLs; real-time publishing; drug approvals; transparency; regulatory disclosure
FDA Pushes Back Agios’ Pyrukynd Thalassemia Decision to December
FDA; Agios; Pyrukynd; thalassemia; PDUFA; decision delay; REMS; hepatocellular injury
FDA Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER
CDER; CBER; rare disease; gene therapy; FDA; submission program; accelerated approval; RDEP; regulatory guidance; rare genetic disorders
FDA Announces New Rare Disease Approval Programme
FDA; rare disease; conditional approval pathway; accelerated approval; biologics; drug review; surrogate endpoints; real-world data; streamlined process
Paul Offit, RFK Jr. Critic, Removed from Key FDA Vaccine Panel
Paul Offit; FDA; vaccine panel; RFK Jr.; removal; vaccine policy; VRBPAC; advisory committee; HHS
Ionis Builds Case for Wider Tryngolza Use in Severe Lipid Disorders Following Positive Phase 3 Results
Ionis Pharmaceuticals; Tryngolza; olezarsen; severe hypertriglyceridemia; lipid disorder; Phase 3 trial; familial chylomicronemia syndrome; acute pancreatitis; FDA; drug indication expansion
Eisai Secures FDA Approval for Leqembi Autoinjector for Maintenance Dosing in Alzheimer’s Patients
Eisai; Leqembi; lecanemab; FDA approval; autoinjector; subcutaneous injection; maintenance dosing; Alzheimer’s disease; at-home treatment; Biogen
Cytokinetics Stock Soars After Positive Phase 3 Data as FDA Approval Decision for Aficamten Nears
Cytokinetics; Aficamten; FDA approval; Phase 3 trial; obstructive hypertrophic cardiomyopathy (oHCM); PDUFA date; REMS; stock performance
Sanofi’s Wayrilz Receives FDA Approval as First BTK Inhibitor for Immune Thrombocytopenia
Wayrilz; rilzabrutinib; FDA approval; immune thrombocytopenia; ITP; BTK inhibitor; Sanofi; LUNA 3 trial
Vinay Prasad Details New Approach to COVID-19 Vaccine Approvals in Recent FDA Memos
Vinay Prasad; COVID-19 vaccine; FDA; vaccine approval; children; Spikevax; regulatory decisions; vaccine safety; evidence; risk-benefit analysis