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
-
Does sickle cell disease increase risk of adverse outcomes following total hip and knee arthroplasty? A nationwide d...Sickle cell disease (SCD) is associated ...
-
Building Capacity to Reduce Stroke in Children With Sickle Cell Anemia in the Dominican Republic: The SACRED TrialThe prevalence of sickle cell anemia (SC...
-
CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell DiseaseThe California Institute for Regenerativ...
-
Crizanlizumab lowers pain crises in at-risk sickle cell patients, ad-hoc trial data showNovartis’ investigational th...
-
Sickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
-
Experimental Gene Therapy Reverses Sickle Cell Disease for YearsA study of an investigational gene thera...
-
Gene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
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.
