Omeros Corporation announced that its investigational drug, zaltenibart (OMS906), has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of complement 3 glomerulopathy (C3G). This ultra-rare and progressive renal disorder primarily affects children and young adults, and currently lacks any approved treatments. The designation underscores the potential of zaltenibart to address a significant unmet medical need in this vulnerable population.
Addressing C3 Glomerulopathy with MASP-3 Inhibition
C3G is characterized by dysregulation of the alternative pathway of complement, often leading to end-stage renal disease within 10 years of diagnosis. Zaltenibart functions as an inhibitor of mannan-binding lectin-associated serine protease-3 (MASP-3), a key activator of the alternative pathway. By blocking MASP-3, zaltenibart prevents the conversion of pro-complement factor D (pro-CFD) to mature CFD, effectively halting the overactivation of the alternative pathway.
"C3G is devastating for children as well as for adults, and our receipt of FDA’s rare pediatric disease designation is a welcome acknowledgment of zaltenibart as a potential therapeutic for this disease that has no approved treatment," stated Gregory A Demopulos, chairman and CEO of Omeros.
Clinical Development and Regulatory Pathway
Omeros is actively advancing zaltenibart through clinical development. Phase 3 clinical trials for zaltenibart in C3G are slated to begin next year. The company is also pursuing zaltenibart for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare and life-threatening blood disease, for which it has already received orphan drug designation from the FDA. Phase 3 studies for zaltenibart in PNH are expected to begin later this quarter.
Priority Review Voucher Program
The Rare Pediatric Disease designation comes with the potential for a valuable incentive. If zaltenibart is approved for C3G in the pediatric population, Omeros will receive a rare pediatric disease priority review voucher from the FDA. This voucher can be used to expedite the review of a New Drug Application (NDA) or Biologics License Application (BLA) for another product, potentially reducing review time by at least four months. The voucher can also be sold to another company.
Expanding Indications and Market Potential
Omeros anticipates expanding the list of targeted indications for zaltenibart and demonstrating its advantages over other alternative pathway inhibitors. The PNH treatment market, for example, was valued at $3.9 billion in 2023 and is projected to reach $10.1 billion in 2032, highlighting the significant commercial opportunity for effective therapies in complement-mediated diseases.
