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
-
Fetal Haemoglobin in Sickle-cell Disease: From Genetic Epidemiology to New Therapeutic StrategiesSickle-cell disease affects millions of ...
-
Statement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Si...bluebird bio, Inc. suspended its clinica...
-
Chronic opioid therapy is more strongly associated with central sensitization in sickle cell disease than clinical p...Central nervous system sensitization (CS...
-
What Should Teens Ask Their Doctors About Hydroxyureahttps://www.youtube.com/watch?v=VZn15uXQ...
-
Ironwood Pharmaceuticals Announces FDA Orphan Drug Designation for Olinciguat for the Treatment of Sickle Cell Disea...Ironwood Pharmaceuticals, Inc., a commer...
-
Sickle Cell Breakthrough: Shauna’s Storyhttps://www.youtube.com/watch?v=Q-kXWV-s...
-
These patients had sickle-cell disease. Experimental therapies might have cured themScientists have long known what causes s...
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.