FDA approves new drug for rare genetic diseaseAugust 19, 2014 @ 3:05 pm
WASHINGTON (AP) -- The Food and Drug Administration has approved a new capsule-based drug to treat Gaucher's disease.
Regulators approved the new drug, Cerdelga, for patients with the Type 1 form of the rare genetic disorder, which causes excess fat to build up in the spleen, liver and bone marrow.
Cerdelga is from Genzyme, the specialty drugmaker that introduced the first drug for Gaucher's disease 20 years ago. The oral treatment could serve as an alternative to Genzyme's best-selling drug Cerezyme, which is given intravenously.
Cerezyme had sales of $935 million last year. The drug costs $300,000 for a year's supply, on average. Genzyme says it will soon release pricing for its new drug.
Type 1 is the most common form of Gaucher's disease and affects an estimated 6,000 people in the U.S.
Copyright © The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.
What Are They Building?
It's hard not to notice a cluster of construction cranes over Seattle's Roosevelt neighborhood
Car Prowl Hot Spots
A new Seattle police study shows where you're most likely to get car prowled
Find holiday events, Santa photo opportunities, and light displays
Please login below with your Facebook, Twitter, Google+ or Disqus account. Existing MyNorthwest account holders will need to create a new Disqus account or use one of the social logins provided below. Thank you.