Rare Diseases
Page 1 • 12 itemsGain critical insights into global orphan drug development & rare disease market trends. Stay ahead in pharma R&D, investment, and regulatory strategy.
Takeda's TAK-881 Meets Primary Endpoint in Phase 2/3 Trial for Primary Immunodeficiency Disease
Takeda announces positive topline results for TAK-881, showing pharmacokinetic comparability to HYQVIA in pivotal Phase 2/3 trial for PID patients.
BioCryst Secures $70M Upfront in European Licensing Deal for Navenibart Hereditary Angioedema Treatment
BioCryst receives $70M upfront plus milestone payments up to $275M for navenibart licensing agreement with Neopharmed Gentili's Irish affiliate in Europe.
Vertex CASGEVY Gene Therapy Reaches 60,000+ Eligible Patients Across 10 Countries as Q1 2026 Results Show Global Expansion
Vertex's CASGEVY, the first approved CRISPR gene therapy, now serves 60,000+ eligible sickle cell disease and beta thalassemia patients across 10 countries.
Stoke Therapeutics Zorevunersen Shows Promise for Dravet Syndrome Treatment Ahead of Q1 2026 Results
Stoke Therapeutics prepares Q1 2026 earnings call featuring Zorevunersen, an investigational antisense therapy targeting Dravet syndrome's underlying cause.
uniQure AMT-130 Gene Therapy Advances Toward UK Approval for Huntington's Disease Treatment
uniQure completes successful UK MHRA pre-submission meeting for AMT-130 Huntington's disease gene therapy, targeting Q3 2026 marketing authorization application.
Alkeus Pharmaceuticals Presents Gildeuretinol Data for Stargardt Disease at ARVO 2026
Alkeus Pharmaceuticals announces clinical data presentation for investigational oral gildeuretinol treating Stargardt disease at ARVO 2026 meeting.
HUTCHMED's Sovleplenib Receives Priority Review and Breakthrough Designation in China for Rare Blood Disorder
HUTCHMED announces NDA acceptance with priority review status for sovleplenib to treat warm antibody autoimmune hemolytic anemia in China.
Ray Therapeutics RTx-015 Receives EMA PRIME Designation for Retinitis Pigmentosa Treatment
Ray Therapeutics' RTx-015 gene therapy receives EMA PRIME designation for retinitis pigmentosa, accelerating regulatory pathway for vision restoration treatment.
X4 Pharmaceuticals Receives European Commission Approval for XOLREMDI (Mavorixafor) as First WHIM Syndrome Treatment in EU
X4 Pharmaceuticals announces European Commission approval for XOLREMDI (mavorixafor), the first authorized treatment for WHIM syndrome patients in the EU.
Norgine Receives European Approval for XOLREMDI (Mavorixafor), First WHIM Syndrome Treatment in EU
Norgine's XOLREMDI becomes the first authorized treatment for WHIM syndrome in Europe, marking a breakthrough for ultra-rare immunodeficiency patients.
Norgine Receives European Commission Approval for XOLREMDI (Mavorixafor) as First WHIM Syndrome Treatment
Norgine's XOLREMDI becomes first authorized treatment for WHIM syndrome in Europe, marking breakthrough for ultra-rare immunodeficiency patients.
Solid Biosciences Receives European Orphan Drug Designation for SGT-003 Duchenne Muscular Dystrophy Treatment
Solid Biosciences secures European Commission orphan drug designation for SGT-003, advancing gene therapy development for Duchenne muscular dystrophy patients.