Catalyst Pharmaceuticals board member to retire
Coral Gables, FL-based Catalyst Pharmaceuticals , Inc. (NASDAQ:CPRX), a $2.69 billion market cap biopharmaceutical company that has delivered an impressive 54% return to shareholders over the past ...
Cure Today3d
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 ...
Taiwan News3d
X4 Pharmaceuticals Announces EMA Validation of Marketing Authorization Application (MAA) for Mavorixafor for the Treatment of WHIM Syndrome
If approved by the EMA, mavorixafor would be the first drug indicated for patients with WHIM syndrome in Europe, a population estimated to be approximately 1,000 people. Earlier this month X4 ...
Pharmaceutical Technology4d
FDA grants ODD status to Zai Lab’s lung cancer treatment
The FDA has granted orphan drug designation (ODD) to Zai Lab’s ZL-1310, aimed at treating small cell lung cancer (SCLC).
Contract Pharma4d
FDA Grants Orphan & Rare Pediatric Designations to Tikun’s FD Treatments
The goal is to treat familial dysautonomia patients with a single injection.
News Medical4d
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 ...
PharmaTimes4d
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 ...
Zydus Life rises after USFDA grants orphan drug status to Usnoflast for ALS treatment
Zydus Lifesciences added 1.10% to Rs 987.60 after the company said that the USFDA had granted orphan drug designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for the treatment of amyotrophic ...
Pharmabiz4d
Zydus’ Usnoflast receives US FDA orphan drug designation to treat amyotrophic lateral sclerosis
Zydus’ Usnoflast receives US FDA orphan drug designation to treat amyotrophic lateral sclerosis: Our Bureau, Mumbai Thursday, January 23, 2025, 12:15 Hrs [IST] Zydus, a leading, ...
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 ...
Zai Lab Receives Orphan Drug Designation from the U.S. FDA for ZL-1310 (DLL3 ADC) for the Treatment of Small Cell Lung Cancer (SCLC)
NDAQ:ZLAB) Zai Lab Receives Orphan Drug Designation from the U.S. FDA for ZL-1310 (DLL3 ADC) for the Treatment of Small Cell ...
BioWorld4d
Tikun programs receive orphan, rare pediatric disease designations
Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the ...