Rejection
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
Outlook Therapeutics Faces New FDA Setback for First Ophthalmic Avastin Formulation
Outlook Therapeutics; Avastin; bevacizumab; Lytenava; wet AMD; FDA rejection; NORSE EIGHT; BLA resubmission; ophthalmic formulation; clinical trial
FDA Rejects PTC’s Vatiquinone Application for Friedreich’s Ataxia, Requests Further Studies
FDA rejection; PTC Therapeutics; vatiquinone; Friedreich’s ataxia; complete response letter; drug efficacy; additional clinical studies
FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful
PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure
Scientists Protest FDA’s Rejection of Replimune’s Tumor Destroying Therapy
Replimune; RP1; FDA rejection; oncolytic immunotherapy; melanoma; clinical trial; IGYNTE study; scientist protest; drug approval; patient heterogeneity
Replimune’s Shares Plunge After FDA Rejects Melanoma Drug Application
Replimune; RP1; FDA rejection; melanoma; cancer drug; Complete Response Letter; clinical trial design; objective response rate; share price drop
FDA Rejects Roche’s Columvi for Earlier-Line Diffuse Large B-Cell Lymphoma
FDA rejection; Columvi; Roche; Genentech; diffuse large B-cell lymphoma; DLBCL; STARGLO study; second-line treatment; Complete Response Letter; gemcitabine; oxaliplatin; accelerated approval; third-line therapy
FDA Rejects Capricor’s Cell Therapy for Duchenne Muscular Dystrophy
FDA rejection; Capricor Therapeutics; deramiocel; Duchenne muscular dystrophy; DMD; cell therapy; cardiomyopathy; clinical data; Phase 3 HOPE-3 trial; Complete Response Letter
FDA Rejects Stealth BioTherapeutics’ Rare Disease Drug but Leaves Door Open for Accelerated Approval
FDA rejection; Stealth BioTherapeutics; elamipretide; Barth syndrome; rare disease; accelerated approval; muscle strength endpoint; phase 2 trial; regulatory delay
Applied Therapeutics’ Rare Disease Drug Fails Another Phase 3 Trial After FDA Rejection
Applied Therapeutics; govorestat; rare disease; phase 3 failure; FDA rejection; classic galactosemia; aldose reductase inhibitor; clinical trial conduct; FDA warning letter