Accelerated – Talk Bio

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

5 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

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

6 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

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

3 weeks 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

4 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

2 months 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

2 months 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