Curated News
By: NewsRamp Editorial Staff
August 21, 2025
ReAlta's GvHD Therapy Gains EMA Orphan Drug Designation
TLDR
- ReAlta's EMA Orphan Drug Designation for RLS-0071 provides market exclusivity and regulatory advantages in Europe for treating steroid-refractory acute GvHD.
- RLS-0071 is a 15-amino-acid peptide that inhibits complement activation at C1 and neutrophil-driven inflammation through MPO and NET formation inhibition.
- This designation accelerates development of a targeted therapy that could save lives by treating rare, life-threatening inflammatory diseases without broad immunosuppression.
- ReAlta's peptide platform mimics human astrovirus mechanisms to uniquely rebalance inflammation in rare diseases like GvHD.
Impact - Why it Matters
This development matters because Graft-versus-Host Disease represents one of the most serious complications following stem cell transplants, with steroid-refractory cases carrying mortality rates as high as 80%. Current treatments rely heavily on broad immunosuppression that compromises patients' entire immune systems, leaving them vulnerable to infections. Pegtarazimod's targeted approach could revolutionize treatment by specifically addressing the destructive inflammatory pathways while preserving protective immune functions. For transplant patients and their families, this represents hope for more effective treatments with fewer side effects. The EMA designation accelerates development timelines and increases the likelihood that this therapy will reach European patients sooner, potentially saving lives and improving quality of life for those facing this devastating condition.
Summary
ReAlta Life Sciences, a clinical-stage biopharmaceutical company, has achieved a significant regulatory milestone with the European Medicines Agency (EMA) granting Orphan Drug Designation to its lead candidate RLS-0071 (pegtarazimod) for the treatment of Graft-versus-Host Disease (GvHD). This designation, supported by preliminary data from the ongoing Phase 2 trial (NCT06343792), recognizes the therapy's potential to address steroid-refractory acute GvHD, a rare and life-threatening condition affecting transplant patients. The EMA's decision follows similar designations from the U.S. FDA, including Orphan Drug and Fast Track Designations granted in August 2024, highlighting global regulatory confidence in pegtarazimod's novel approach.
Pegtarazimod represents a breakthrough in inflammatory disease treatment through its dual-targeting mechanism that modulates both complement and neutrophil-mediated inflammation. Unlike broad immunosuppressive therapies, this 15-amino-acid peptide selectively inhibits complement activation at C1 while targeting myeloperoxidase activity and neutrophil extracellular trap formation—key drivers of tissue damage in GvHD. The ongoing Phase 2 trial is currently enrolling hospitalized patients with moderate to severe steroid-refractory aGvHD across clinical sites in the United States, Germany, and Spain, with additional data expected in 2026. Company leadership, including CEO David Marek and Chief Medical Officer Kenji Cunnion, emphasizes that this targeted approach preserves beneficial immune function while addressing the specific pathways responsible for the most difficult-to-treat lower gastrointestinal manifestations of the disease.
The Orphan Drug Designation provides ReAlta with substantial benefits including reduced regulatory fees, clinical protocol assistance, and potential market exclusivity in the European Union upon approval. This development marks a crucial step toward addressing the urgent unmet need in aGvHD treatment, particularly for patients who fail to respond to standard steroid therapies. The company's EPICC peptide platform, derived from human astrovirus research, demonstrates innovative science that could transform treatment paradigms for multiple rare inflammatory conditions beyond GvHD, including hypoxic ischemic encephalopathy and COPD exacerbations.
Source Statement
This curated news summary relied on content disributed by citybiz. Read the original source here, ReAlta's GvHD Therapy Gains EMA Orphan Drug Designation
