Alnylam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll
Score another early win at the FDA for Alnylam.
The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.
An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 148,200+ biopharma pros reading Endpoints daily — and it's free.