Google
Trusted Resources: News & Events
Latest announcements and gatherings
Sancilio Pharmaceuticals Company announces Altemia™ receives orphan drug designation from the European Medicines Agency for the treatment of pediatric patients with Sickle Cell Disease (SCD)
Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced that the European Commission has granted Orphan Designation in the European Union to Altemia, the Company’s novel oral formulation being developed for the treatment of SCD in pediatric patients. This approval follows a positive opinion in February 2018 from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).
Orphan Designation is granted by the European Commission to drugs that are intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating rare diseases. Rare diseases are those defined as having a prevalence of not more than five per 10,000 population in the European Union. The designation potentially qualifies the sponsor for ten years of marketing exclusivity upon approval, as well as fee reduction for various centralized activities, including applications for marketing authorization, inspections and protocol assistance.
+myBinderRelated Content
-
Association of Antibiotic Choice With Hospital Length of Stay and Risk Factors for Readmission in Patients With Sick...Study objective: We determine the assoc...
-
Opioid utilization by pregnant women with sickle cell disease and the risk of neonatal abstinence syndromeBACKGROUND:Pregnant women with sickle ce...
-
World’s Largest Sickle Cell Disease Stem Cell Library UnderwayScientists in Boston are creating a stem...
-
Gene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
-
Possible Cure for Sickle Cell Found in ParisHematologists, sickle cell patients, and...
-
SCDAA News Advisory: Partial Hold on Gene Therapy TrialOn December 20, the FDA placed a partial...
-
Facebook Live: Stem Cells and Sickle Cell Diseasehttps://www.youtube.com/watch?v=IPzNx8i_...
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.