Infrequent
AstraZeneca Onshores Rare Disease Portfolio as Part of $50B US Investment Pledge
AstraZeneca; rare disease portfolio; onshoring; US investment; biomanufacturing; $50 billion pledge; Virginia facility; Maryland investment; R&D expansion
FDA Breakthrough Designations Lead to Priority Reviews, Report Finds
FDA; breakthrough therapy designation; priority review; drug approval; expedited pathway; biotechnology innovation; rare diseases; oncology; CDER; regulatory science
Arrowhead Secures Maiden FDA Approval for Redemplo in Rare Genetic Metabolic Disorder (FCS)
Arrowhead Pharmaceuticals; FDA approval; Redemplo; plozasiran; familial chylomicronemia syndrome; rare genetic disorder; RNA interference; TRiM platform; triglyceride reduction; first commercial drug
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