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' CASGEVY Reaches 60,000+ Eligible Patients Across 10 Countries Following Global Approvals
CRISPR Therapeutics reports CASGEVY now approved in 10 countries for severe sickle cell disease and beta thalassemia, reaching over 60,000 eligible patients worldwide.
Clene's CNM-Au8 Gets FDA Green Light for Accelerated Approval Pathway in ALS Treatment
FDA confirms Clene's CNM-Au8 ALS drug can proceed via accelerated approval pathway using neurofilament light biomarker data, with NDA filing expected Q3 2026.
Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen in McCune-Albright Syndrome
Atossa Therapeutics gains FDA Rare Pediatric Disease designation for (Z)-endoxifen treating McCune-Albright syndrome, qualifying for Priority Review Voucher.
Novartis Receives Health Canada Approval for Fabhalta Oral Treatment for C3G Patients
Health Canada approves Novartis' Fabhalta (iptacopan) oral treatment for adults with complement 3 glomerulopathy, offering new hope for rare kidney disease patients.
Cytokinetics to Report Aficamten Phase 3 Trial Results for Non-Obstructive Hypertrophic Cardiomyopathy May 5, 2026
Cytokinetics announces topline results from pivotal ACACIA-HCM Phase 3 trial of aficamten for non-obstructive hypertrophic cardiomyopathy on May 5, 2026.
FDA Approves Incyte's Jakafi XR Extended-Release Tablets for Myelofibrosis and Blood Disorders
Incyte receives FDA approval for once-daily Jakafi XR (ruxolitinib) extended-release tablets for myelofibrosis, polycythemia vera, and GVHD treatment.
Mirum Pharmaceuticals VISTAS Study Results for Volixibat in Primary Sclerosing Cholangitis Due May 4, 2026
Mirum Pharmaceuticals will announce topline results from the VISTAS study of volixibat for primary sclerosing cholangitis treatment on May 4, 2026.
NIKTIMVO (Axatilimab) Approved in Australia as First-in-Class Chronic Graft-Versus-Host Disease Treatment
Australia's TGA approves NIKTIMVO (axatilimab), a breakthrough first-in-class therapy for chronic graft-versus-host disease under priority review.
BioMarin VOXZOGO Shows Long-Term Benefits for Achondroplasia in New Clinical Data at PES 2026
BioMarin presents new VOXZOGO data showing long-term benefits on arm span, bone health and growth in children with achondroplasia at PES 2026 meeting.
FDA Approves Incyte's Jakafi XR Extended-Release Tablets for Myelofibrosis, Polycythemia Vera Treatment
Incyte receives FDA approval for Jakafi XR (ruxolitinib) extended-release tablets, offering improved dosing convenience for rare blood cancer patients.
Neurocrine Biosciences CRENESSITY Shows Sustained Benefits in Two-Year Pediatric CAH Study
CRENESSITY (crinecerfont) demonstrates durable hormone control and reduced glucocorticoid exposure in pediatric congenital adrenal hyperplasia patients.
Rhythm Pharmaceuticals' IMCIVREE Receives European Commission Approval for Acquired Hypothalamic Obesity
IMCIVREE becomes first therapy approved in both US and Europe for acquired hypothalamic obesity, targeting adults and children 4+ with hypothalamic injury.