Accelerated – Talk Bio

FDA Announces First Recipients of Makary’s National Priority Voucher Program, Expanding Scope for Accelerated Drug Reviews

1 week ago talkbio0Tagged Accelerated, CNPV, domestic drug manufacturing, Drug Approval, Martin Makary, National Priority Voucher, pilot program, Priority Review, public health crisis, recipients, United States Food and Drug Administration

FDA; National Priority Voucher; CNPV; Martin Makary; priority review; accelerated drug approval; 2025 pilot program; voucher recipients; public health crisis; domestic drug manufacturing

FDA Delays Denali’s Hunter Syndrome Therapy Decision by Three Months

2 weeks ago talkbio0Tagged Accelerated, Alfa, amendment, Approved, Biologics License Application, Clinical pharmacology discipline, Deferred, Denali, Mucopolysaccharidosis II, review extension, tividenofusp, United States Food and Drug Administration

FDA delay; Denali Therapeutics; Hunter syndrome; tividenofusp alfa; MPS II; Biologics License Application (BLA); review extension; clinical pharmacology; major amendment; accelerated approval

FDA Approves Stealth BioTherapeutics’ Forzinity as First Treatment for Barth Syndrome After Rejection and Delay

1 month ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Abnormality of mitochondrial metabolism, Accelerated, Approved, Elamipretide, Forzinity, Infrequent, Stealth BioTherapeutics, United States Food and Drug Administration

FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; rare disease; mitochondrial dysfunction; accelerated approval

FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome

1 month ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Accelerated, Approved, Clinical Trials, Elamipretide, Forzinity, Infrequent, Mitochondrial Diseases, Pediatric Disorder, Stealth BioTherapeutics, United States Food and Drug Administration

FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial

Daiichi and Merck’s Lung Cancer ADC Achieves 48% Response Rate in Pivotal Trial

2 months ago talkbio0Tagged Accelerated, Antibody-Drug Conjugates, Approved, Breakthrough Therapy, CD276 gene, Co., Daiichi, Deruxtecan, Frequency of Responses, IDeate-Lung01, Identifier, ifinatamab, Overall Survival, Progression-Free Survival, Sankyo, Small cell carcinoma of lung, therapeutic autologous dendritic cells, United States Food and Drug Administration

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 Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER

2 months ago talkbio0Tagged Accelerated, Approved, CBER, CDER, gene therapy, Hereditary Diseases, Infrequent, RDEP, regulatory guidance, submission program, United States Food and Drug Administration

CDER; CBER; rare disease; gene therapy; FDA; submission program; accelerated approval; RDEP; regulatory guidance; rare genetic disorders

FDA Announces New Rare Disease Approval Programme

2 months ago talkbio0Tagged Accelerated, Approved, Biological Factors, conditional approval pathway, drug review, Endpoints, Infrequent, real-world data, streamlined process, Surrogate, United States Food and Drug Administration

FDA; rare disease; conditional approval pathway; accelerated approval; biologics; drug review; surrogate endpoints; real-world data; streamlined process

FDA Grants Wegovy 3rd Indication: First GLP-1 Approved for MASH with Moderate-to-Advanced Liver Scarring

2 months ago talkbio0Tagged Accelerated, Approved, Fibrosis, Liver, MASH, noncirrhotic, Nordisk, Product containing glucagon-like peptide 1 receptor agonist (product), semaglutide, United States Food and Drug Administration, Wegovy

Wegovy; FDA approval; MASH; semaglutide; GLP-1 receptor agonist; liver fibrosis; Novo Nordisk; ESSENCE trial; noncirrhotic; accelerated approval

Boehringer Ingelheim breaks into oncology as FDA grants accelerated approval to Hernexeos (zongertinib) for HER2 TKD–mutated NSCLC

3 months ago talkbio0Tagged Accelerated, Approved, Beamion, Boehringer Ingelheim, competition, Confirmatory Trial, Enhertu, erbB-2 Receptor, Frequency of Responses, HER2-mutant, Hernexeos, Identifier, LUNG-1, Mutation, Non-Small Cell Lung Carcinoma, non-squamous NSCLC, Oncomine Dx Target Test, oral targeted therapy, Priority Review, Requirement, TKD, United States Food and Drug Administration, Zongertinib

Boehringer Ingelheim; Hernexeos; zongertinib; FDA approval; accelerated approval; non-squamous NSCLC; HER2 TKD mutations; Oncomine Dx Target Test; Beamion LUNG-1; objective response rate; first oral targeted therapy for HER2-mutant NSCLC; priority review; breakthrough therapy designation; confirmatory trial requirement; competition with Enhertu

FDA Grants Accelerated Approval to Jazz’s Dordaviprone (Modeyso), the First Treatment for Ultra-Rare Brain Tumor

3 months ago talkbio0Tagged Accelerated, Approved, Brain Neoplasms, Chimerix acquisition, Confirmatory Trial, Dordaviprone, midline glioma, Modeyso, pediatric brain cancer, United States Food and Drug Administration

Dordaviprone; Modeyso; FDA approval; H3 K27M-mutant diffuse midline glioma; ultra-rare brain tumor; Jazz Pharmaceuticals; accelerated approval; pediatric brain cancer; Chimerix acquisition; confirmatory trial