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.
NMPA Conditional Approval: What You Need to Know About China's Expedited Drug Pathway
Learn about China's NMPA Conditional Approval process, designed to expedite access to critical medications like XYZ for cancer patients.
NMPA Accelerated Approval Pathway: Impact on Innovative Drug Market Entry in China
The NMPA Accelerated Approval Pathway significantly enhances the speed of innovative drug market entry in China, particularly for critical cancer treatments.
PMDA SAKIGAKE Designation: Accelerating Innovative Therapies in Japan
The PMDA SAKIGAKE Designation fast-tracks innovative therapies, such as XYZ Drug for cancer, enhancing patient access to groundbreaking treatments in Japan.
PMDA SAKIGAKE Designation: Accelerating Innovation in Japan's Pharma Sector
The PMDA SAKIGAKE Designation is transforming Japan's pharmaceutical landscape, expediting the development of innovative drugs like XYZ for cancer treatment.
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.
PMDA SAKIGAKE Designation: Accelerating Rare Disease Therapy Approvals in Japan
The PMDA SAKIGAKE Designation fast-tracks the approval process for rare disease therapies, exemplified by XYZ Drug for ABC indication in Japan.