Google
Trusted Resources: News & Events
Latest announcements and gatherings
Imara’s IMR-687 for SCD Receives FDA Rare Pediatric Disease Designation
The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to IMR-687, Imara’s drug candidate for the treatment of sickle cell disease (SCD). The investigational therapy has also been granted orphan drug status by the FDA.
Rare pediatric disease designation is granted to drugs that show promise for rare and serious or life-threatening diseases in U.S. patients, primarily age 18 years or younger. The designation provides incentives to advance the development of rare disease drugs, including access to the FDA’s expedited review and approval programs.
“People living with sickle cell disease have limited treatment options for its devastating effects,” James McArthur, PhD, Imara’s president and CEO, said in a news release. “The FDA’s decision reflects its commitment to working with innovators and the patient community toward advancing safe and effective treatments for children suffering from the rare and damaging pediatric manifestations of the disease.”
+myBinderRelated Content
-
HABIT, a Randomized Feasibility Trial to Increase Hydroxyurea Adherence, Suggests Improved Health Related Quality of...Objectives: To examine the effect of a...
-
FDA Approves First Targeted Therapy to Treat Patients with Painful Complication of Sickle Cell DiseaseToday, the U.S. Food and Drug Administra...
-
Initial results from a cohort in a phase 2a study (GBT440-007) evaluating adolescents with sickle cell disease treat...Background: Sickle cell disease (SCD), ...
-
Today’s Faces of Sickle Cell Disease: John Tisdale, MDFor many years, John Tisdale has been wo...
-
NIH Launches new Collaboration to Develop Gene-Based Cures for Sickle Cell Disease and HIV on Global ScaleThe National Institutes of Health plans ...
-
Preventing Strokes in Children with Sickle Cell Diseasehttps://www.youtube.com/watch?v=k6h7cb7S...
-
Sisterly bond behind sickle-cell curehttps://www.youtube.com/watch?v=9S-xVGsR...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.