United States Food and Drug Administration
Capricor Claims FDA Misinterpreted Efficacy Data Leading to Rejection of DMD Therapy
Capricor Therapeutics; FDA rejection; deramiocel; Duchenne muscular dystrophy (DMD); efficacy data misinterpretation; clinical trial endpoints; HOPE-2 trial; non-parametric test; statistical significance; cell therapy
LB Pharmaceuticals Raises $285M in Upsized IPO to Fund Late-Stage Schizophrenia Drug Development
LB Pharmaceuticals; IPO; schizophrenia; LB-102; amisulpride; Phase 3 clinical trial; bipolar depression; biotech fundraising; CNS drugs; FDA approval
LB Pharmaceuticals Files for $228M IPO to Fund Phase III Schizophrenia Drug
LB Pharmaceuticals; IPO; Phase III trial; schizophrenia; LB-102; biotech; CNS therapeutics; bipolar depression; Alzheimer’s psychosis; amisulpride; FDA; Nasdaq
Daiichi and Merck’s Lung Cancer ADC Achieves 48% Response Rate in Pivotal Trial
Daiichi Sankyo; Merck & Co.; ifinatamab deruxtecan; I-DXd; B7-H3; antibody-drug conjugate; ADC; small cell lung cancer; SCLC; IDeate-Lung01; objective response rate; accelerated approval; progression-free survival; overall survival; FDA Breakthrough Therapy Designation
FDA Shifts Approach on GLP-1 Compounding: Announces Consumer Green List Instead of Immediate Crackdown
FDA; GLP-1; compounding; green list; drug shortages; semaglutide; tirzepatide; import alert; consumer safety; regulation
Recent Developments in Logistical Coordination for Decentralized Clinical Trials (DCTs)
Decentralized Clinical Trials; Logistics; FDA Guidance 2024; Remote Patient Monitoring; Supply Chain Management; Digital Trial Platforms
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