Forma Therapeutics has secured orphan drug status from the European Commission (EC) for its FT-4202 to treat sickle cell disease (SCD).
SCD, which is a common single-gene disorder, is estimated to affect over 70,000 in the EU-27 and up to 100,000 people in the US.
The status was provided based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA).
FT-4202 is a novel selective red blood cell (RBC) pyruvate kinase-R (PKR) activator designed to serve as a disease-modifying therapy to treat SCD.
The multimodal approach enables FT-4202 to work upstream and activate the RBCs’ natural PKR activity to decrease 2,3-DPG levels. The downstream activity of FT-4202 increases ATP levels to enhance RBC health and survival.
In April this year, the company secured rare paediatric disease and orphan drug designations from the US Food and Drug Administration (FDA) to treat patients with SCD.
At present, the company is recruiting SCD patients in a randomised, placebo-controlled and multi-centre phase 1 study to assess the safety and pharmacokinetics/pharmacodynamics of FT-4202.
Forma Therapeutics president and CEO Frank Lee said: “Without effective treatment, the sickle cell can affect all organs over time and lead to substantial suffering for those born with this genetic disease.
“We embrace the potential this designation may provide as we prepare to initiate a global registrational Phase 2/3 trial with FT-4202 in the first quarter of 2021.”
Forma Therapeutics is involved in the research, development and commercialisation of novel therapeutics to treat rare haematologic diseases and cancers.