ReAlta Life Sciences has received orphan drug designation from the European Medicines Agency (EMA) for its investigational therapy RLS-0071 (pegtarazimod) in graft-versus-host disease (GvHD). The designation follows preliminary results from the company’s ongoing phase 2 trial in steroid-refractory acute GvHD (aGvHD) patients.
Pegtarazimod is a synthetic peptide designed to rebalance the body’s inflammatory response by targeting both neutrophil- and complement-mediated pathways. It works by selectively inhibiting complement activation, myeloperoxidase activity and neutrophil extracellular trap formation – processes that drive tissue damage when donor immune cells attack host tissue after a stem cell transplant.
The phase 2 study is enrolling hospitalised patients in the US, Germany and Spain with moderate to very severe steroid-refractory aGvHD. Early findings from the first patient cohort supported ReAlta’s application to the EMA, which grants orphan status to medicines targeting rare conditions affecting fewer than five in 10,000 people in the EU. Benefits include reduced regulatory fees, clinical advice and up to ten years of market exclusivity if approved.
ReAlta has already secured orphan and fast track designations for pegtarazimod in aGvHD from the US Food and Drug Administration. The EMA decision validates the drug’s potential and would help accelerate development in Europe.
Founded in 2018, ReAlta is advancing its pipeline of therapies for rare and acute inflammatory conditions from its bases in Virginia in the US and Puerto Rico.