Infrequent – Talk Bio

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

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

2 weeks 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 weeks 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

Amylyx Discontinues Rare Disease Program After Relyvrio Fails to Show Benefit Over Placebo

3 weeks ago talkbio0Tagged AMX0035, Amylyx, Amyotrophic Lateral Sclerosis, Clinical trial failure, Discontinuation (procedure), disease program, Infrequent, Placebos, Progressive, Relyvrio, Supranuclear paralysis

Amylyx; Relyvrio; AMX0035; progressive supranuclear palsy; ALS; clinical trial failure; rare disease program; placebo; discontinuation

Regeneron Reports Phase 3 Success for Cemdisiran in Rare Disease Generalized Myasthenia Gravis

3 weeks ago talkbio0Tagged Alnylam, cemdisiran, Infrequent, Myasthenia Gravis, Generalized, Regeneron, regulatory submission, RNA drug, RNA, Small Interfering, United States

Regeneron; Phase 3 trial; cemdisiran; generalized myasthenia gravis; rare disease; RNA drug; siRNA; Alnylam Pharmaceuticals; U.S. regulatory submission

Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug

4 weeks ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, access program, Drug Approval, Elamipretide, Infrequent, resubmission, Stealth BioTherapeutics, United States Food and Drug Administration

Stealth BioTherapeutics; FDA resubmission; rare disease; Barth syndrome; elamipretide; drug approval; expanded access program

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

4 weeks 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

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

1 month 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

Novartis antibody ianalumab clinches another Phase 3 win in rare blood disease (ITP)

1 month ago talkbio0Tagged Autoimmune, Autoimmune thrombocytopenia, B-cell depletion, ianalumab, Infrequent, Inosine Triphosphate, Novartis, Phase 3, platelet levels, Psychological inhibition, regulatory submissions, Sjögren 's disease, TNFRSF13C protein, human, treatment failure, VAY736

Novartis; ianalumab; VAY736; primary immune thrombocytopenia; ITP; rare disease; Phase 3; time to treatment failure; safe platelet levels; eltrombopag; autoimmune; B-cell depletion; BAFF-R inhibition; Sjögren’s disease; regulatory submissions

Y-mAbs Therapeutics Acquired by SERB Pharmaceuticals in $412 Million Buyout Following Strategic Review

1 month ago talkbio0Tagged all-cash tender, Danyelza, Infrequent, Merger, Neoplasms, pharmaceutical acquisition, Serbs, strategic review, Y-mAbs

Y-mAbs Therapeutics; SERB Pharmaceuticals; buyout; Danyelza; rare oncology; merger; pharmaceutical acquisition; strategic review; all-cash tender offer