Hosted on MSN10h
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.
Pharmaceutical Technology16h
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 Pharma16h
FDA Grants Orphan & Rare Pediatric Designations to Tikun’s FD Treatments
The goal is to treat familial dysautonomia patients with a single injection.
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome ...
ophthalmologytimes19h
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 ...
PharmaTimes21h
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 ...