PDUFA – Talk Bio

Sanofi’s multiple sclerosis drug tolebrutinib flunks Phase 3 in PPMS and faces another FDA PDUFA delay

7 days ago talkbio0Tagged BTK inhibitor, Clinical trial failure, Deferred, Mississippi (geographic location), Multiple Sclerosis, non-relapsing secondary progressive, nrSPMS, Parts per million (qualifier value), PDUFA, Progressive, Regulatory Setback, Review [Publication Type], sanofi, Tolebrutinib, United States Food and Drug Administration

Sanofi; tolebrutinib; multiple sclerosis; primary progressive multiple sclerosis; PPMS; non-relapsing secondary progressive MS; nrSPMS; Phase 3 PERSEUS trial; PDUFA delay; FDA review; BTK inhibitor; clinical trial failure; regulatory setback

FDA Delays Decision on Ascendis’ Dwarfism Drug by Three Months

4 weeks ago talkbio0Tagged Achondroplasia, Approved, Ascendis Pharma, CNP gene, Deferred, Dwarfism, navepegritide, PDUFA, post-marketing requirement, TransCon, United States Food and Drug Administration

FDA; Ascendis Pharma; TransCon CNP; navepegritide; achondroplasia; dwarfism; PDUFA; post-marketing requirement; delay; approval

Nuvalent Announces FDA Acceptance of New Drug Application for Zidesamtinib in TKI Pre-treated Advanced ROS1-positive NSCLC

1 month ago talkbio0Tagged acceptance, ARROS-1 trial, Breakthrough Therapy, Clinical Nurse Specialists, Identifier, New Drug Application, Non-Small Cell Lung Carcinoma, Nuvalent, orphan drug designation, PDUFA, Penetration, pre-treated, Protein-tyrosine kinase inhibitor, ROS1-positive, United States Food and Drug Administration, zidesamtinib

Nuvalent; zidesamtinib; FDA acceptance; New Drug Application (NDA); ROS1-positive NSCLC; tyrosine kinase inhibitor (TKI) pre-treated; ARROS-1 trial; PDUFA date; CNS penetration; breakthrough therapy designation; orphan drug designation

FDA Extends Review Period for Rhythm Pharmaceuticals’ Imcivree (Setmelanotide) in Acquired Hypothalamic Obesity

1 month ago talkbio0Tagged Agonist, amendment, goal, Hypothalamic structure, Imcivree, MC4R protein, human, PDUFA, Phase 3 trial, review extension, Rhythm Pharmaceuticals, setmelanotide, United States Food and Drug Administration

Imcivree; Rhythm Pharmaceuticals; FDA; review extension; acquired hypothalamic obesity; setmelanotide; PDUFA goal date; Phase 3 trial; major amendment; MC4R agonist

FDA Delays Decision on Sanofi’s Oral Multiple Sclerosis Drug to December 2025

3 months ago talkbio0Tagged BTK inhibitor, Clinical action, December 2025, drug review, Multiple Sclerosis, nrSPMS, PDUFA, sanofi, Tolebrutinib, United States Food and Drug Administration

FDA action date; Sanofi; tolebrutinib; multiple sclerosis; BTK inhibitor; nrSPMS; drug review delay; December 2025; PDUFA

FDA Pushes Back Agios’ Pyrukynd Thalassemia Decision to December

4 months ago talkbio0Tagged Agios, decision delay, Liver damage, PDUFA, Pyrukynd, REMS, Thalassemia, United States Food and Drug Administration

FDA; Agios; Pyrukynd; thalassemia; PDUFA; decision delay; REMS; hepatocellular injury

Cytokinetics Stock Soars After Positive Phase 3 Data as FDA Approval Decision for Aficamten Nears

4 months ago talkbio0Tagged Aficamten, Approved, Cytokinetics, Hypertrophic obstructive cardiomyopathy, PDUFA, Performance, REMS, United States Food and Drug Administration

Cytokinetics; Aficamten; FDA approval; Phase 3 trial; obstructive hypertrophic cardiomyopathy (oHCM); PDUFA date; REMS; stock performance

FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121

4 months ago talkbio0Tagged clemidsogene lanparvovec, Deferred, gene therapy, IDS gene, Infrequent, Mucopolysaccharidosis II, PDUFA, Regenxbio, RGX-121, United States Food and Drug Administration

FDA delay; PDUFA extension; Regenxbio; Hunter syndrome; clemidsogene lanparvovec; RGX-121; gene therapy; Mucopolysaccharidosis II; IDS gene; rare disease