Category: Clinical Trials
FDA approves AMVUTTRA for treatment of hATTR amyloidosis with polyneuropathy
BREAKING NEWS: The U.S. Food and Drug Administration today approved Alnylam Pharmaceuticals AMVUTTRATM, a novel therapy for the treatment of hereditary transthyretin (hATTR) amyloidosis with polyneuropathy. This approval comes after positive results from the readout of HELIOS-A Phase III study...
Vutrisiran receives Fast Track Designation from FDA
Alnylam has submitted a new drug application (NDA) to the FDA for vutrisiran, which could bring another treatment option to patients with ATTR amyloidosis. Vutrisiran received “Fast Track” Designation, which is a process designed to expedite the review of drugs...
Report From the Amyloidosis Forum Scientific Workshop
The Amyloidosis Forum, a Public Private Partnership with ARC and FDA, hosted the workshop on 22nd January, as part of an ongoing process to develop novel endpoints for clinical trial in AL amyloidosis. In 2020, the Amyloidosis Forum launched a...
First treatment for AL Amyloidosis approved in US
Daratumumab has just been granted approval by the FDA for the treatment of AL Amyloidosis in the United States. Today marks a major milestone and an achievement that has been years in the making. Janssen applied for FDA approval for...
Early Findings from Novel CRISPR Therapy Show Promise for hATTR Amyloidosis
July 7, 2021 CRISPR technology offers a cutting-edge approach to gene-editing. New clinical findings from Intellia’s Phase 1 trial have demonstrated for the first time that CRISPR can be administered by infusion to target specific cells within the body. (more…)