Nov 25, 2024
− Prescription Drug User Fee Act (PDUFA) Date Set for March 23, 2025 −
Nov 25, 2024
− Prescription Drug User Fee Act (PDUFA) Date Set for March 23, 2025 −
11.22.2024 at 7:12 PM EST
- Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes
- Attruby demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date:
CAMBRIDGE, Mass., Nov. 16, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also known as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for ATTR amyloidosis.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 30, 2024-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced detailed results from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).