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News about "Orphan Drug Designation "
Partner Therapeutics Wins FDA Approval for BIZENGRI in NRG1+ Cholangiocarcinoma
Partner Therapeutics receives US Food and Drug Administration (FDA) approval for BIZENGRI, the first targeted therapy for NRG1 fusion-positive cholangiocarcinoma, expanding use across NSCLC and pancreatic cancer.
Orphan Drug Designation | 12/05/2026 | By News Bureau
Rznomics Secures US FDA RMAT Designation for Liver Cancer Gene Therapy Candidate RZ-001
The FDA has granted Regenerative Medicine Advanced Therapy designation to RZ-001, an RNA-based gene therapeutic for hepatocellular carcinoma, accelerating its clinical development and regulatory review pathway.
Orphan Drug Designation | 11/05/2026 | By News Bureau
ImmunoForge Unveils LMT15 Platform to Transform CNS Drug Delivery
The clinical-stage biotech firm ImmunoForge is advancing its next-generation blood-brain barrier shuttle technology, combining enhanced brain permeability with long-acting formulations to accelerate therapies for CNS and rare diseases.
Orphan Drug Designation | 24/04/2026 | By News Bureau
Abbisko's Irpagratinib Secures EMA Orphan Drug Tag for Liver Cancer
European regulator grants rare disease designation to FGFR4 inhibitor, boosting its clinical development and commercial prospects in hepatocellular carcinoma.
Orphan Drug Designation | 01/04/2026 | By News Bureau
EU Grants Conditional Approval to Rezurock for Chronic GVHD Treatment
European Commission authorises belumosudil for patients aged 12 plus with limited treatment options, marking a significant advance in managing a life-threatening post-transplant condition.
Orphan Drug Designation | 01/04/2026 | By News Bureau
GSK plc Secures Japan Orphan Drug Status for Ris-Rez in Small-Cell Lung Cancer
Japan’s health ministry grants orphan drug designation to GSK’s investigational ADC Ris-Rez for SCLC, supported by early clinical data showing durable responses in advanced-stage patients.
Orphan Drug Designation | 24/03/2026 | By News Bureau | 119
Aisa Pharma presented Phase II RECONNOITER trial results showing AISA-021 improved attack-free days and reduced duration of Raynaud’s attacks in systemic sclerosis patients at the World Systemic Sclerosis Congress.
Orphan Drug Designation | 10/03/2026 | By News Bureau
Japan Grants Orphan Drug Designation to Sanofi's Rilzabrutinib
Sanofi’s rilzabrutinib has received Orphan Drug Designation in Japan for IgG4-related disease (IgG4-RD), supported by positive phase-II study results. The decision marks the therapy’s third global orphan designation in this indication, underscoring Sanofi’s continued focus on advancing treatments for rare immune-mediated diseases.
Orphan Drug Designation | 02/03/2026 | By News Bureau | 105
GSK's Linerixibat Granted Priority Review in China for Cholestatic Pruritus in PBC
China’s drug regulator has accepted GSK’s linerixibat for priority review to treat cholestatic pruritus in primary biliary cholangitis, supported by Phase III data showing significant and sustained itch relief.
Orphan Drug Designation | 27/02/2026 | By News Bureau | 153
FDA Grants Orphan Drug Status to HanchorBio's HCB101 for Gastric Cancer
The Orphan Drug Status designation to HanchorBio supports development of next-generation CD47–SIRPα immunotherapy for advanced gastric adenocarcinoma across HER2-positive and HER2-negative subtypes.
Orphan Drug Designation | 16/02/2026 | By News Bureau | 135
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