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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.
education & researchOpioid utilization by pregnant women with sickle cell disease and the risk of neonatal abstinence syndromeBACKGROUND:Pregnant women with sickle ce...
videos & visualsBlood Transfusions: What You Need to Know and Do (Part 3: IRON OVERLOAD)https://www.youtube.com/watch?v=aXURPZav...
education & researchRed blood cell transfusion to treat or prevent complications in sickle cell disease: An overview of Cochrane reviewsBackground: Globally, sickle cell disea...
news & eventsSolution to 50-year-old mystery could lead to gene therapy for common blood disordersIn a landmark study that could lead to n...
Community CenterPossible Cure for Sickle Cell Found in ParisHematologists, sickle cell patients, and...
news & eventsInvestigational Therapy Altemia Achieves Main Endpoints in Pediatric Sickle Cell TrialSancilio Pharmaceuticals says its invest...
news & eventsBoston Children’s Hospital receives grant for sickle cell disease researchThe Bill and Melinda Gates Foundation aw...
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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.