Posted by Michael Wonder on 07 Sep 2017
Global Blood Therapeutics receives rare paediatric disease designation from FDA for GBT440 for treatment of sickle cell disease
5 September 2017 - Global Blood Therapeutics today announced that the U.S. FDA has granted rare paediatric disease designation to GBT440 for the treatment of sickle cell disease.
GBT440 is being developed as a potentially disease-modifying therapy for sickle cell disease (SCD).
GBT440 is being developed as an oral, once-daily therapy for patients with SCD. GBT440 works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerise, GBT believes GBT440 blocks polymerization and the resultant sickling of red blood cells. With the potential to restore normal haemoglobin function and improve oxygen delivery, GBT believes that GBT440 may potentially modify the course of SCD. In recognition of the critical need for new SCD treatments, the U.S. FDA has granted GBT440 fast track, orphan drug and rare paediatric disease designations for the treatment of patients with SCD. The EMA has included GBT440 in its Priority Medicines (PRIME) program, and the European Commission has designated GBT440 as an orphan medicinal product for the treatment of patients with SCD.
