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...
Amyloidosis Forum highlighted as an exemplary model to enhance product development
The Amyloidosis Forum, a public-private partnership initiated between the Amyloidosis Research Consortium and FDA, was highlighted recently during FDA Rare Disease Day Virtual Public Meeting on March 4th as an exemplary model to enhance product development using a public-private partnership...
Remembering Dr. Merrill Benson
The Amyloidosis Research Consortium extends our deepest, heartfelt condolences to the family and friends of Dr. Merrill Benson, world renowned amyloidosis expert, who passed away on September 11, 2021. He was 83.
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...
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...