Approved – Talk Bio

FDA Approves Darzalex Faspro for High-Risk Smoldering Multiple Myeloma

11 hours ago talkbio0Tagged Approved, AQUILA study, daratumumab, Darzalex Faspro, Early Intervention (Education), high-risk, Progression-Free Survival, Smoldering myeloma, United States Food and Drug Administration

FDA approval; Darzalex Faspro; high-risk smoldering multiple myeloma; AQUILA study; early intervention; progression-free survival; daratumumab; hyaluronidase-fihj

Sarepta’s Duchenne Muscular Dystrophy Drugs Fail Confirmatory Trial, Company Pursues Full FDA Approval Regardless

4 days ago talkbio0Tagged Accelerated, AMONDYS, Approved, Clinical trial failure, Confirmatory Trial, ELEVIDYS, Muscular Dystrophy, Duchenne, Statistical Significance, United States Food and Drug Administration

Sarepta Therapeutics; Duchenne muscular dystrophy; confirmatory trial; AMONDYS 45; VYONDYS 53; Elevidys; FDA approval; clinical trial failure; accelerated approval; statistical significance

UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease: Kygevvi for Thymidine Kinase 2 Deficiency (TK2d)

4 days ago talkbio0Tagged Approved, Breakthrough Therapy, doxecitine, doxribtimine, Drugs, Orphan, Infrequent, Kygevvi, Mitochondrial Diseases, Pediatric Disorder, Priority Review, Thymidine kinase 2 deficiency, TK2d, UCB, United States Food and Drug Administration

UCB; FDA approval; Kygevvi; mitochondrial disease; Thymidine Kinase 2 deficiency; TK2d; doxecitine; doxribtimine; rare diseases; Orphan Drug; Breakthrough Therapy; Priority Review; Rare Pediatric Disease

uniQure Faces Delay as FDA Walks Back Accelerated Approval for Huntington’s Gene Therapy

5 days ago talkbio0Tagged Accelerated, AMT-130, Approved, Clinical Trials, gene therapy, Huntington 's disease, Regulatory Setback, UniQure, United States Food and Drug Administration

uniQure; AMT-130; Huntington’s disease; gene therapy; FDA; accelerated approval; regulatory setback; clinical trial

Dyne Therapeutics’ z-rostudirsen Shows Durable Efficacy, Supporting Positive Regulatory Outlook

1 week ago talkbio0Tagged Accelerated, Approved, Biological Markers, Clinical Trials, DELIVER (veterinary product), Dyne Therapeutics, Effectiveness, Functional improvement, Muscular Dystrophy, Duchenne, regulatory approval, z-rostudirsen

Dyne Therapeutics; z-rostudirsen; DELIVER trial; Duchenne muscular dystrophy (DMD); durable efficacy; regulatory approval; biomarkers; functional improvement; accelerated approval

BridgeBio Shifts Filing Plans After Phase 3 Success in Rare Disease Drug BBP-418

2 weeks ago talkbio0Tagged Acoramidis, Approved, BBP-418, Biological Markers, BridgeBio, commercial, disease drug, File (record), Infrequent, Limb-girdle muscular dystrophy type 2A, NDA, New Drug Application, Phase 3 success, Ribitol, United States Food and Drug Administration

BridgeBio; BBP-418; Phase 3 success; limb-girdle muscular dystrophy type 2I/R9; FDA filing; rare disease drug; NDA (New Drug Application); ribitol; full approval; biomarker; acoramidis; commercial strategy

Bayer Receives FDA Approval for Lynkuet, a Novel Non-Hormonal Drug for Menopausal Hot Flashes

2 weeks ago talkbio0Tagged Approved, Bayer, Elinzanetant, Hot flushes, Lynkuet, Menopause, NK1/NK3 receptors, Non-Hormonal, United States Food and Drug Administration, vasomotor symptom

Bayer; FDA approval; Lynkuet; elinzanetant; hot flashes; menopause; non-hormonal; NK1/NK3 receptors; vasomotor symptoms

FDA Approves Syndax’s First-in-Class Menin Inhibitor with Black-Box Warning; Updates on GSK’s ADC Deal

2 weeks ago talkbio0Tagged Acute leukemia, Antibody-Drug Conjugates, Approved, black-box warning, Chromosomal translocation, differentiation syndrome, KMT2A gene, Leukemia, Myelocytic, Acute, Menin Inhibitor, oncology drug approval, Revuforj, revumenib, SmithKline Beecham, Syndax, United States Food and Drug Administration

Syndax; Revuforj; revumenib; FDA approval; menin inhibitor; black-box warning; acute leukemia; KMT2A translocation; differentiation syndrome; acute myeloid leukemia (AML); GSK; antibody-drug conjugate (ADC); oncology drug approval