GBT Scoops Up Orphan Drug Status for Sickle Cell Candidate

The FDA has granted orphan drug status to a sickle cell disease candidate from Global Blood Therapeutics.

GBT440 could be the first disease-modifying treatment in the genetic blood condition that causes organ damage. A once-daily oral medication, the candidate stimulates oxygenation of hemoglobin, averting polymerization and thus the sickling of red blood cells.

GBT hopes to launch a pivotal Phase 3 trial in adult SCD patients this year.