orphan drug designation

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

1 month 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

2 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

2 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