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.
Contract Pharma36m
FDA Grants Orphan & Rare Pediatric Designations to Tikun’s FD Treatments
The goal is to treat familial dysautonomia patients with a single injection.
Moneylife1h
Main Indices stabilise, Broader Market Bounces Back, FII Selling Continues: Nifty, Sensex: Thursday Closing Report
On Thursday, 2119 stocks advanced, 1842 declined and 106 remained unchanged, with an advance decline ratio of 1.15 on the ...
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome
Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, ...
The Pharma Letter2h
GlobalData tracks 40 years of innovation in FDA review designations
Regulatory authorities, particularly the US Food and Drug Administration (FDA), have played a crucial role in accelerating and incentivizing the development of innovative treatments through review ...
ophthalmologytimes3h
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 ...
PharmaTimes5h
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 ...
Daily6h
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 ...
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 ...
Pharmabiz7h
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)
Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan ...