Alnylam Pharmaceuticals announced that the FDA has provided orphan drug designation to ALN-TTR02 for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant clinical manifestations of transthyretin (TTR)-mediated amyloidosis (ATTR).

Transthyretin (TTR)-mediated amyloidosis (ATTR) is a hereditary, systemic disease caused by mutations in the TTR gene. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.

ALN-TTR02 is a systemically delivered RNAi therapeutic being developed for the treatment of ATTR. It is formulated using lipid nanoparticle technology.

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