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.
CRISPR Therapeutics Reports Q1 2026 Results as CASGEVY Gene Therapy Gains Global Regulatory Approval
CRISPR Therapeutics reports Q1 2026 financial results highlighting CASGEVY's approval in 10+ countries for sickle cell disease and beta thalassemia treatment.
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.
Biosimilar Uptake EU: Market Impact Post-Patent Expirations & HTA Regulation
This article examines how biosimilar adoption in the EU is reshaping the market landscape for biologics like Adalimumab after patent expirations and under HTA regulations.
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.
EU Pharmaceutical Legislation Reform: Impact on Orphan Drug Market Exclusivity
This article examines how recent EU pharmaceutical legislation reforms could reshape market exclusivity for orphan drugs, affecting access to treatments for rare diseases.
EU Clinical Trials Regulation Impact on Rare Disease Drug Development
This article examines the impact of the EU Clinical Trials Regulation on the development of drugs for rare diseases, focusing on challenges and opportunities for treatments.
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.