orphan drug designation

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

3 weeks 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

Jazz and Zymeworks Bispecific Antibody Zanidatamab Delivers Practice-Changing Results in Stomach Cancer Study

3 weeks ago talkbio0Tagged Antibodies, Bispecific, erbB-2 Receptor, gastroesophageal adenocarcinoma, Jazz, Malignant neoplasm of stomach, orphan drug designation, Overall Survival, Pharmacotherapy, Phase 2 trial, Phase 3, practice-changing, Progression-Free Survival, Zanidatamab, Zymeworks

Jazz Pharmaceuticals; Zymeworks; zanidatamab; bispecific antibody; HER2; gastroesophageal adenocarcinoma; stomach cancer; phase 2 trial; phase 3 HERIZON-GEA-01; overall survival; progression-free survival; chemotherapy; practice-changing; Orphan Drug designation

Diamyd Medical’s Pivotal Phase 3 Type 1 Diabetes Trial Clears Final Safety Review Ahead of Early Readout in March 2026

4 weeks ago talkbio0Tagged Accelerated, approval pathway, Data Safety, Diabetes Mellitus, Insulin-Dependent, DIAGNODE-3, Diamyd Medical, DSMB, fast-track designation, Genotype, HLA DR3-DQ2, interim readout, Monitoring Board, orphan drug designation, Phase 3 trial, precision therapy, safety review

Diamyd Medical; Type 1 Diabetes; Phase 3 trial; DIAGNODE-3; safety review; Data Safety Monitoring Board (DSMB); interim readout; March 2026; HLA DR3-DQ2 genotype; Orphan Drug Designation; Fast Track Designation; accelerated approval pathway; precision therapy

Ionis Heads to FDA as Antisense Therapy Aces Pivotal Study in Rare Neurological Disease

3 months ago talkbio0Tagged access program, Alexander Disease, Antisense Oligonucleotides, Fast Track, Identifier, Improvement, Infrequent, Ionis Pharmaceuticals, nervous system disorder, orphan drug designation, Phase I – III study, Speed, United States Food and Drug Administration, Zilganersen

Ionis Pharmaceuticals; zilganersen; antisense oligonucleotide; Alexander disease; pivotal Phase I–III study; FDA Fast Track designation; rare neurological disease; expanded access program; walk speed improvement; Orphan Drug designation

Servier Supercharges Neuro Pipeline with $450M Deal for Fragile X Syndrome Candidate

3 months ago talkbio0Tagged Acquisition, Bioscience, deal, Fragile X Syndrome, Infrequent, Kaerus, KER-0193, orphan drug designation, pediatric drug designation, Phase 2 trial, Servier

Servier; Kaerus Bioscience; KER-0193; Fragile X syndrome; neurology pipeline; $450 million deal; orphan drug designation; rare pediatric drug designation; Phase 2 trial; acquisition

FDA Grants Full Approval to Precigen’s Papzimeos for Rare Disease RRP

4 months ago talkbio0Tagged Approved, Episodic, gene therapy, Immunotherapy, Infrequent, orphan drug designation, Papzimeos, Precigen, Recurrent respiratory papillomatosis, RRP, United States Food and Drug Administration

Precigen; Papzimeos; FDA approval; recurrent respiratory papillomatosis (RRP); gene therapy; immunotherapy; rare disease; Orphan Drug designation

Stoke Therapeutics Advances Dravet Syndrome Drug to Phase III Trials After FDA Lifts Partial Clinical Hold

1 year ago talkbio0Tagged Activator, Infantile Severe Myoclonic Epilepsy, orphan drug designation, pediatric disease designation, Phase 3 Clinical Trials, SCN1A gene, Stoke, United States Food and Drug Administration, Zorevunersen

Stoke Therapeutics, Dravet syndrome, Phase III trials, FDA, zorevunersen, SCN1A activator, orphan drug designation, rare pediatric disease designation