Alnylam makes RNAi history again as patisiran gets approved in Europe; Vertex buys into UK-based machine learning group
→ Just days after Alnylam landed a historic FDA OK on patisiran — and quickly launched it as Onpattro — the European Commission has come through with its own marketing authorization. The drug, which treats hereditary ATTR amyloidosis by silencing messenger RNA and blocking the production of TTR protein, is the first RNAi therapy to be approved in Europe. It’s also a likely blockbuster-to-be, with Clarivate pegging 2022 projected sales at $1.21 billion even though the disease afflicts only about 30,000 people worldwide. Inotersen, a rival product from Akcea and Ionis, was approved in Europe in July.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.
