News Medical5d
What is an Orphan Drug? A Guide to Rare Disease Therapies
Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...
March Biosciences Receives FDA Orphan Drug Designation for MB-105, a First-in-Class CD5 CAR-T Cell Therapy, for T-Cell Lymphoma
Company prepares to advance to Phase 2 clinical trial in early 2025HOUSTON, Jan. 28, 2025 (GLOBE NEWSWIRE) -- March Biosciences (March Bio), an ...
Cure Today4d
FDA Approves GRAFAPEX/Fludarabine Before alloHSCT in AML and MDS
The FDA has approved GRAFAPEX™ and fludarabine as a preparative regimen in acute myeloid leukemia or myelodysplastic syndrome ...
PharmaTimes5d
Zydus announces USFDA Orphan Drug Designation for Usnoflast in ALS treatment
Zydus has announced that the USFDA has granted Orphan Drug Designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for ...
Japan grants orphan drug status to HAE treatment
CAMBRIDGE, Mass. & SALISBURY, England - KalVista Pharmaceuticals, Inc. (NASDAQ: NASDAQ:KALV) has received Orphan Drug ...
devdiscourse6d
Zydus Strides Forward with Orphan Drug for ALS
Zydus Lifesciences announced that Usnoflast, a treatment for amyotrophic lateral sclerosis (ALS), has received Orphan Drug Designation from the US FDA. This designation supports the development of ...
Zai Lab Gets FDA Orphan Drug Designation For ZL-1310 To Treat Small Cell Lung Cancer
Zai Lab Limited (ZLAB) announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation or ODD to ZL-1310, a ...
Nuvalent: A First Look
Nuvalent, Inc. shares have fallen approximately 30% after promising updates on its two lead NSCLC candidates rallied the ...
Zydus receives USFDA Orphan Drug Designation for Usnoflast to treat ALS
Zydus Lifesciences announced that the USFDA granted Orphan Drug Designation to Usnoflast, a novel oral NLRP3 inhibitor, for ...