Eleva, a pioneer in unlocking difficult-to-produce biologics based on a breakthrough manufacturing platform, announced today that the European Commission has granted the Orphan Drug Designation for Factor H (CPV-104), the company’s recombinant version of human complement Factor H, to treat C3 Glomerulopathy (C3G). Eleva is preparing a first clinical study in C3G and expects to commence dosing patients in H1 2025.

“Today’s 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) program. The positive opinion by the EMA is also a clear validation for the comprehensive preclinical data sets we have generated for our program,” commented Andreas Schaaf, Ph.D., Chief Scientific Officer of Eleva. “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.”

The Orphan Drug Designation (ODD) in the EU was granted by the European Commission based on a positive opinion issued previously by the EMA Committee for Orphan Medicinal Products (COMP). The program is focused on the development of drugs that have shown the potential to provide significant benefit to patients suffering from rare, life-threatening diseases. Following market approval, the ODS designation would provide Eleva with ten years of market exclusivity and provides special incentives, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees.

C3G represents a rare renal disease caused by the dysregulation of the complement system, a central part of the body’s immune defense. Naturally occurring complement regulators such as Factor H can provide a pro-resolutive option for restoring the balance of the complement system. Preclinical data sets recently published in Frontiers in Immunology point towards Factor H (CPV-104)’s ability to act as a functional analogue of human Factor H, support normalization of serum C3 levels and the rapid degradation of C3 deposits in the kidney.