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.
Ascidian Therapeutics Completes ACDN-01 Dose Escalation in STELLAR Phase 1/2 Trial for Stargardt Disease
Ascidian Therapeutics completes adult dose escalation for ACDN-01 in Stargardt disease Phase 1/2 STELLAR trial, expands to pediatric patients.
Arrowhead Pharmaceuticals Receives TGA Approval for REDEMPLO (Plozasiran) in Australia for Rare Metabolic Disorder
Arrowhead Pharmaceuticals' REDEMPLO (plozasiran) receives Australian TGA approval for familial chylomicronemia syndrome, expanding global access.
Ironwood Pharmaceuticals Survey Reveals Critical Gaps in Short Bowel Syndrome TPN Treatment at DDW 2026
Ironwood Pharmaceuticals presents DDW 2026 survey findings highlighting unmet needs in total parenteral nutrition therapy for short bowel syndrome patients.
Arrowhead Pharmaceuticals Receives TGA Approval for REDEMPLO (Plozasiran) in Australia for Rare Genetic Disorder
Arrowhead Pharmaceuticals gains Australian TGA approval for REDEMPLO (plozasiran), expanding global access for familial chylomicronemia syndrome patients.
BioMarin's PALYNZIQ Receives FDA Approval for Adolescent PKU Patients 12 and Older
BioMarin announces FDA approval of PALYNZIQ for adolescents 12+ with phenylketonuria (PKU), expanding treatment options for rare genetic disorder patients.
FDA Approves Travere's FILSPARI for FSGS Treatment in Adults and Children 8+
Travere Therapeutics receives FDA approval for FILSPARI to reduce proteinuria in FSGS patients aged 8 and older, expanding rare kidney disease treatment options.
Argo Biopharma Doses First Patient in Phase I Trial of siRNA Therapy BW-50218 for TTR-Related Disease
Argo Biopharma begins Phase I clinical trial of BW-50218, an siRNA therapeutic targeting transthyretin protein for rare disease treatment in Australia.
BioMarin Completes $3 Billion Acquisition of Amicus Therapeutics, Expands Rare Disease Portfolio with Galafold and Pompe Therapies
BioMarin's acquisition of Amicus adds globally approved Galafold for Fabry disease and POMBILITI/OPFOLDA combination for Pompe disease to its rare disease pipeline.
Spinogenix Launches Phase 2b/3 CLARITY Trial of SPG601 for Fragile X Syndrome Treatment
Spinogenix initiates CLARITY Phase 2b/3 trial testing SPG601, a first-in-class oral therapy targeting BK channels for Fragile X Syndrome in male patients.
Calluna Pharma Completes CAL101 Phase 2 Enrollment for Idiopathic Pulmonary Fibrosis Treatment
Calluna Pharma enrolls 161 patients in Phase 2 AURORA study of CAL101 for IPF treatment, six months ahead of schedule with topline data expected Q1 2027.
Argo Biopharma Begins Phase II Trials of BW-40202 siRNA Therapy for Rare Kidney Diseases
Argo Biopharma doses first patients in Phase II trials of BW-40202, an investigational siRNA therapy targeting complement factor B for PNH and IgAN treatment.
NMPA Priority Review Pathway: Accelerating Oncology & Rare Disease Drug Approvals
The NMPA Priority Review Pathway significantly speeds up the approval process for oncology and rare disease drugs, ensuring timely access to life-saving therapies.