FT-4202 and ARU-1801 granted Orphan Drug Status in EU for sickle cell disease

The European Medicines Agency (EMA) has recently granted the designation of orphan drug to two pharmaceutical agents for the treatment of sickle cell disease (SCD); FT-4202 (Forma Therapeutics) and ARU-1801 (Aruvant). FT-4202 is a novel selective red blood cell (RBC) pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy for the treatment of SCD. The end result of this therapy is to increase haemoglobin levels in order to improve RBC health and function, and to decrease painful vaso-occlusive crises. Forma is currently enrolling patients with SCD in a randomized, placebo-controlled, multi-center Phase 1 study to evaluate the safety and pharmacokinetics/pharmacodynamics (PK/PD) of FT-4202 (NCT03815695), and plans to initiate a global registrational Phase 2/3 trial with FT-4202 in the first quarter of 2021. ARU-1801 is an investigational lentiviral gene therapy that aims to restore normal RBC function through increased levels of foetal haemoglobin in adult patients. Preliminary clinical data from the Phase 1/2 MOMENTUM study demonstrates that ARU-1801 can achieve durable reductions in disease burden with a reduced intensity conditioning regimen. The FDA previously granted Orphan Drug and Rare Pediatric Disease Designations to both FT-4202 and ARU-1801 for the treatment of patients with SCD. More info: Forma press release, Aruvant press release