FDA approves new drug for rare genetic disease

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 This material may not be published, broadcast, rewritten or redistributed.


Top Stories

  • Back In Boom
    Seahawks' defense returns to its dominant form against Arizona

  • 'Tis the Ski Season
    Cascade ski areas are crossing their fingers for an early start to the season
ATTENTION COMMENTERS: We've changed our comments, but want to keep you in the conversation.
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.
comments powered by Disqus
Sign up for breaking news e-mail alerts from MyNorthwest.com
In the community
Do you know an exceptional citizen who has impacted and inspired others?
KIRO Radio and WSECU would like to recognize six oustanding citizens this year. Nominate them to be recognized and to receive a $2,000 charitable grant.