Infrequent
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
New Confirmatory Study Suggests Progress in Applied Therapeutics–FDA Alignment for Rare Disease Drug
Applied Therapeutics; govorestat; CMT-SORD; rare disease; FDA; confirmatory study; clinical trial; regulatory alignment
Sanofi Injects $625M Into Biotech Investment Arm to Accelerate Innovation
Sanofi; Sanofi Ventures; biotech investment; $625M cash infusion; digital health; immunology; rare diseases; neurology; venture capital; cell/gene therapy; portfolio expansion
Ionis Heads to FDA as Antisense Therapy Aces Pivotal Study in Rare Neurological Disease
Ionis Pharmaceuticals; zilganersen; antisense oligonucleotide; Alexander disease; pivotal Phase I–III study; FDA Fast Track designation; rare neurological disease; expanded access program; walk speed improvement; Orphan Drug designation
FDA Approves Stealth BioTherapeutics’ Forzinity as First Treatment for Barth Syndrome After Rejection and Delay
FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; rare disease; mitochondrial dysfunction; accelerated approval
FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome
FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial
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