Infrequent
FDA Unveils ‘Plausible Mechanism Pathway’ to Accelerate Personalized Therapies, Inspired by Baby KJ
FDA; personalized therapies; gene editing; plausible mechanism pathway; Baby KJ; rare diseases; CRISPR; regulatory innovation
Cogent Hits Unprecedented Efficacy in Rare Stomach Cancer, Clearing Path to FDA
Cogent Biosciences; bezuclastinib; unprecedented efficacy; rare stomach cancer; gastrointestinal stromal tumor (GIST); FDA submission; second-line treatment; objective response rate; progression-free survival
UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease: Kygevvi for Thymidine Kinase 2 Deficiency (TK2d)
UCB; FDA approval; Kygevvi; mitochondrial disease; Thymidine Kinase 2 deficiency; TK2d; doxecitine; doxribtimine; rare diseases; Orphan Drug; Breakthrough Therapy; Priority Review; Rare Pediatric Disease
Researchers Publish Roadmap of FDA Interactions After Baby KJ’s Groundbreaking Gene Editing Therapy, as FDA Signals New Pathway for Patient-Specific Treatments
Baby KJ; CRISPR gene editing; CPS1 deficiency; FDA approval pathway; personalized medicine; rare disease; regulatory roadmap; platform trials; N-of-1 therapies; rare disease gene therapies
No Recent News on BridgeBio’s Thyroid Disorder Phase 3 Win
BridgeBio; Phase 3; Rare Diseases; Thyroid Disorder
BridgeBio Shifts Filing Plans After Phase 3 Success in Rare Disease Drug BBP-418
BridgeBio; BBP-418; Phase 3 success; limb-girdle muscular dystrophy type 2I/R9; FDA filing; rare disease drug; NDA (New Drug Application); ribitol; full approval; biomarker; acoramidis; commercial strategy
Inhibrx to Seek FDA Approval in 2026 After Strong Phase 2 Data for Bone Cancer Drug Ozekibart
Inhibrx; FDA submission; ozekibart; INBRX-109; chondrosarcoma; bone cancer; phase 2 clinical trial; progression-free survival; death receptor 5; biologics license application; rare cancer; liver toxicity
Novo Nordisk Acquires Rights to Omeros’ MASP-3 Inhibitor Zaltenibart
Novo Nordisk; Omeros; Zaltenibart; MASP-3 Inhibitor; Rare Blood and Kidney Disorders
Fortress Biotech and Sentynl Therapeutics Fail to Secure FDA Approval for CUTX-101 in Rare Pediatric Menkes Disease
Fortress Biotech; Sentynl Therapeutics; FDA Complete Response Letter; CUTX-101; Menkes disease; rare pediatric disease; manufacturing deficiencies; drug approval setback
Star Therapeutics Raises $125M as Bleeding Disorder Therapy Enters Phase III
Star Therapeutics; Series D financing; bleeding disorders; VGA039; Phase III clinical trial; biotech funding; rare diseases; antibody therapy