Eleva, a clinical-stage biopharmaceutical company, has been assigned Orphan Drug Designation (ODD) for its Factor H therapy CPV-104 to treat complement-3 glomerulopathy.
The novel drug is a recombinant version of the classic human complement Factor H, and plans are currently underway to commence clinical studies using CPV-104 in patients with C3G.
The company aims to begin trials in early 2025.
What is C3G
Complement-3 glomerulopathy is a rare disease of the kidneys that is categorised by the dysregulation of the complement system — a critical part of the body’s immune defence.
During disease pathology, there is an abnormally high level of the immune protein C3, which can accumulate in the kidneys and impact its functioning.
Factor H, a naturally occurring regulator of the complement system, can assist in restoring its balance, so targeting it for therapeutic purposes could significantly improve patient outcomes.
Achieving Orphan Drug Designation
ODD for the Factor H therapy was granted in the EU by the European Commission due to an opinion previously issues by the EMA Committee for Orphan Medicinal Products (COMP).
Since C3G is a rare and potentially fatal disease, there are little treatment options for patients. The designation aims to accelerate the therapeutic through the clinical trial process.
If the drug is successful in trials, the ODS designation would provide Eleva with ten years of market exclusivity, as well as providing special incentives such as eligibility for protocol assistance and regulatory fee exemptions.
“This news marks an important milestone for Eleva’s R&D and regulatory teams, as the Orphan Drug Designation adds significant value to the Factor H (CPV-104) programme. The positive opinion by the EMA is also a clear validation for the comprehensive preclinical data sets we have generated for our programme,” commented Chief Scientific Officer of Eleva, Andreas Schaaf.
He continues: “Overall, Factor H (CPV-104) continues to progress steadily towards clinical studies and is emerging rapidly as a potential first-in-class solution for C3G and potentially other complement-related disorders.”