Pediatric Disorder

UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease: Kygevvi for Thymidine Kinase 2 Deficiency (TK2d)

3 weeks ago talkbio0Tagged Approved, Breakthrough Therapy, doxecitine, doxribtimine, Drugs, Orphan, Infrequent, Kygevvi, Mitochondrial Diseases, Pediatric Disorder, Priority Review, Thymidine kinase 2 deficiency, TK2d, UCB, United States Food and Drug Administration

UCB; FDA approval; Kygevvi; mitochondrial disease; Thymidine Kinase 2 deficiency; TK2d; doxecitine; doxribtimine; rare diseases; Orphan Drug; Breakthrough Therapy; Priority Review; Rare Pediatric Disease

Fortress Biotech and Sentynl Therapeutics Fail to Secure FDA Approval for CUTX-101 in Rare Pediatric Menkes Disease

2 months ago talkbio0Tagged biotech, Complete, CUTX-101, deficiencies, Drug Approval, Fortress, Infrequent, Manufacturing, Menkes disease, Pediatric Disorder, Sentynl Therapeutics, United States Food and Drug Administration

Fortress Biotech; Sentynl Therapeutics; FDA Complete Response Letter; CUTX-101; Menkes disease; rare pediatric disease; manufacturing deficiencies; drug approval setback

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