The Food and Drug Administration on Friday approved a landmark rare disease treatment — the first to rely on a Nobel-prize-winning technique known as RNA interference, which silences disease-causing genes.
The approval is a major accomplishment for Cambridge, Mass.-based Alnylam, which will be marketing the drug, patisiran, as Onpattro and which has been working to bring an RNAi-based therapy to market for more than a decade.
The agency gave patisiran a relatively narrow green light. Its only indication is for neuropathy, or nerve damage. Unlike the European regulator’s initial opinion, the FDA’s press release announcing the approval made no mention of Onpattro’s possible effect on heart damage, which is also a common symptom of the disease, known as hereditary transthyretin amyloidosis, or hATTR.
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There are volumes of cases of Friedreich’s ataxia in Louisiana. How do we get the word out to neurologist here?