Contract Pharma1h
FDA Grants Orphan & Rare Pediatric Designations to Tikun’s FD Treatments
The goal is to treat familial dysautonomia patients with a single injection.
News Medical1d
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 ...
PharmaTimes6h
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 ...
Pharmaceutical Technology1h
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).
ophthalmologytimes4h
FDA grants fast track designation to N-acetylcysteine amide tablets from Nacuity Pharmaceuticals
The NPI-001 (N-acetylcysteine amide) tablets are a proprietary investigational therapy for the treatment of patients with ...
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 ...
Japan grants orphan drug status to HAE treatment
CAMBRIDGE, Mass. & SALISBURY, England - KalVista Pharmaceuticals, Inc. (NASDAQ: NASDAQ:KALV) has received Orphan Drug ...
Pharmabiz8h
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, ...
Daily7h
Zydus Usnoflast gets USFDA Orphan Drug Designation for amyotrophic lateral sclerosis
Ahmedabad: Zydus, a discovery-based, global pharmaceutical company has announced that the U.S. Food and Drug Administration ...
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 ...
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 ...
Intellia doses first subject in trial of NTLA-2002 for hereditary angioedema
The HAELO trial will assess the therapy’s safety and efficacy in 60 adult subjects with Type I or Type II HAE.