Drugs, Orphan
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
Unlocking Japan’s Potential in Rare Disease Drug Development: Recent Progress and Challenges (2025)
rare disease; Japan; orphan drug; drug lag; innovation; regulatory approval; pharmaceutical partnerships; clinical trials
Regeneron Prepares U.S. Submission for Garetosmab After Breakthrough Phase III Result in Ultra-Rare FOP
Regeneron; garetosmab; FOP; fibrodysplasia ossificans progressiva; Phase III; OPTIMA trial; bone lesion prevention; rare disease; FDA submission; orphan drug
FDA Grants Dupixent (Dupilumab) Eighth Approval: Now Cleared for Bullous Pemphigoid
Dupixent; Dupilumab; Sanofi; Regeneron; bullous pemphigoid; FDA approval; type 2 inflammation; autoimmune disease; priority review; orphan drug
Regeneron Leads $66M Series B for Actio Biosciences’ Rare Disease Therapeutics
Regeneron; Actio Biosciences; rare diseases; Series B financing; KCNT1 inhibitor; TRPV4 inhibitor; ABS-1230; ABS-0871; Charcot-Marie-Tooth disease; epilepsy; orphan drug; clinical trials
Senate Drops Orphan Cures Act from Trump’s Tax Bill, Dealing Setback to Biopharma
Orphan Cures Act; orphan drugs; Trump tax bill; biopharma; Senate; IRA price negotiations; rare diseases; Big Beautiful Bill; healthcare legislation; reconciliation bill
Projected $270 Billion Orphan Drug Market by 2028: A Comprehensive Analysis of Leading Players J&J, Vertex, and Roche
Orphan drugs, rare diseases, J&J, Vertex, Roche, market growth, 2028, Evaluate