Alnylam Pharmaceuticals said this week that the US Food and Drug Administration has given orphan drug status to the company's investigational transthyretin-mediated amyloidosis ALN-TTR02 as a treatment for familial amyloidotic polyneuropathy, a common manifestation of the disease.
The designation provides a drugmaker with, among other things, extended market exclusivity for its product, tax credits, and marketing incentives.
ALN-TTR02 recently entered phase II testing (GSN 6/7/2012).