United States Food and Drug Administration
UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data
UniQure, Huntington’s disease, gene therapy, AMT-130, FDA, RMAT designation, Phase I/II clinical trials, neurodegenerative disorder, neurofilament light (NfL), disease progression, composite Unified Huntington’s Disease Rating Scale (cUHDRS).
FDA Approves Eli Lilly’s Alzheimer’s Drug Donanemab, Offering New Treatment Option
Alzheimer’s disease, Eli Lilly, FDA approval, donanemab, Kisunla, anti-amyloid monoclonal antibody, cognitive decline, amyloid plaques, treatment options
FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease Over Manufacturing Concerns
FDA, Rocket Pharmaceuticals, Gene Therapy, Leukocyte Adhesion Deficiency-I (LAD-I), Manufacturing Concerns
J&J, Legend’s Phase III Win Cements Carvykti as Earlier-Line Multiple Myeloma Treatment
Carvykti, Multiple Myeloma, Phase III, CARTITUDE-4, FDA Approval, BCMA-Targeted Therapy, Johnson & Johnson, Legend Biotech
FDA Approves Lilly’s Donanemab for Alzheimer’s Treatment Amid Manufacturing Concerns
FDA Approval, Donanemab, Alzheimer’s Disease, Eli Lilly, Manufacturing Issues, Amyloid-Targeting Drug
Supreme Court Overturns Chevron Doctrine, Limiting FDA’s Regulatory Authority
Chevron doctrine, FDA, regulatory authority, Supreme Court, administrative law, agency interpretations, statutory ambiguity
FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease, Requests Additional Manufacturing Information
FDA, Rocket Pharmaceuticals, Gene Therapy, Leukocyte Adhesion Deficiency-I (LAD-I), Manufacturing Information, Complete Response Letter
Merck and Daiichi Sankyo’s ADC Pact Hits Regulatory Setback in FDA Rejection
Merck, Daiichi Sankyo, Antibody-Drug Conjugate (ADC), Patritumab Deruxtecan, FDA Rejection, Regulatory Setback, HER3 Protein, Non-Small Cell Lung Cancer (NSCLC)
Lykos Faces Setback as ICER Concludes Insufficient Evidence for MDMA-Based PTSD Treatment
Lykos Therapeutics, MDMA, PTSD, ICER, Insufficient Evidence, Clinical Trials, FDA Approval