Alnylam announced that the FDA has granted Fast Track designation to patisiran (ALN-TTR02) for the treatment of transthyretin (TTR)-familial amyloid polyneuropathy(FAP). ALN-TTR02 is a systemically delivered RNAi therapeutic formulated using lipid nanoparticle technology.
The company recently announced Phase 2 data which showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%. Knockdown of TTR was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein.
Alnylam has initiated an open label extension (“OLE”) study for patients that participated in the Phase 2 study and the APOLLO Phase 3 trial of patisiran in ATTR patients with FAP.
For more information call (617) 551-8200 or visit Alnylam.com.