FDA Approves First Targeted Therapy to Treat Patients with Painful Complication of Sickle Cell Disease

On November 15, 2019, the U.S. Food and Drug Administration approved Adakveo (crizanlizumab-tmca), a treatment to reduce the frequency of vaso-occlusive crisis – a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells – for patients age 16 years and older.

“Hope has never been higher for people living with sickle cell disease and their families and supporters, with a pipeline of new treatments on the horizon, like the one being approved today, and several initiatives underway to better utilize current tools in the battle against the painful and deadly blood disorder,” said Acting FDA Commissioner Adm. Brett P. Giroir, M.D. “The opportunity before us in the coming months and years is profound and historic.”

Read more >>

Share this post

CONTACT US
KEEP IN TOUCH
Sign up for our email list, or follow us on social media, to stay in-the-know about the Maryland Sickle Cell Disease Association
Scroll to Top

Breaking News

FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

On December 8, 2023, “The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy."