The U.S. FDA has granted Effient (prasugrel hydrochloride) Orphan Drug Designation for the treatment of sickle cell disease (SCD).  Effient is manufactured and marketed by global pharmaceutical company Eli Lilly.  It is an anti-coagulant indicated for prevention of blood clots in patients with acute coronary syndrome undergoing angioplasty.  SCD is a rare hereditary blood disorder caused by mutation in the beta-globin gene.  It is characterized by sickle-like shaped red blood cells under certain circumstances and may lead to various complications, such as anemia and a variety of medical crises.  Currently, there is no effective treatment for this rare disease.  A multicenter phase II study of prasugrel in adult patients with SCD has shown a decrease in platelet activation biomarkers and a trend toward decreased pain.  Recently, Eli Lilly in collaboration with Daiichi Sankyo has completed a phase II trial in pediatric SCD patients.

 

References:

  1. https://clinicaltrials.gov/ct2/show/NCT01476696?term=prasugrel&rank=8
  2. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm171497.htm
  3. http://www.jhoonline.org/content/6/1/17