According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food and Drug Administration (FDA). This investigational treatment is in development as a treatment for polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients. Alnylam is focused on developing RNA interference based treatments for a wide variety of diseases.
About HATTR Amyloidosis
HATTR Amyloidosis is a disease which is characterized by the build up of amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. The disease is caused by a heritable genetic mutation. Symptoms of the illness include swelling, heart failure, irregular heartbeat, shortness of breath, fatigue, weight loss, easy bruising and bleeding, stroke, lung problems, enlarged liver, and changes in skin color. Treatment approaches for hATTR amyloidosis includes liver transplant; This can actually cure the disease, but the procedure carries substantial risks and side effects. This form of amyloidosis has a better prognosis than some of the more common types with some patients surviving for over a decade. It is estimated to affect around 50,000 people worldwide. To learn more about amyloidosis, click here.
About Fast Track Designation
Fast Track designation is designed to increase the speed of development of a promising drug that is in development to treat a currently unmet medical need or treat a serious, life-threatening disease. Alternatively, the drug in question must show potential advantages over currently available treatments. A drug that earns Fast Track designation is eligible for several different benefits. These benefits include rolling review (allowing a New Drug Application to be reviewed per section, speeding up the process), more frequent in-person and written communications with the FDA related to development, and potential eligibility for Priority Review and Accelerated Approval if other requirements are met.
So far, vutrisiran has demonstrated favorable safety characteristics in phase 1 testing and is currently undergoing further evaluation in phase 3 clinical trials, which have recently completed enrollment. The experimental therapy has also been given Orphan Drug status in the US and EU for the treatment of hATTR amyloidosis. Hopefully, the results of the ongoing studies will be promising.