FDA Approves Onpattro, a Novel RNAi Therapeutic to Treat Polyneuropathy in Adults
FDA Approves Onpattro, a Novel RNAi Therapeutic to Treat Polyneuropathy in Adults
Shots:
- Alnylam’s siRNA molecule Onpattro receives FDA approval for fatal genetic disorder polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR)
- Approval is based on results from Ph III APOLLO study involving 19 countries with the ratio of 2:1 of Onpattro vs Placebo once Q3W for 18mos showing improved QoL
- Alnylam is expected to receive CHMP opinion by Sep’18, with expected regulatory filings in other markets, including Japan in H2’18
Click here to read full press release/ article | Ref: US FDA | Image: Forbes