Omeros' Zaltenibart Receives FDA Rare Pediatric Disease Designation for C3 Glomerulopathy
Key Insights
The FDA has granted Rare Pediatric Disease designation to Omeros' zaltenibart (OMS906) for treating C3 glomerulopathy (search) (C3G (search)), a rare kidney disorder in children.
Omeros plans to initiate Phase 3 clinical trials for zaltenibart in C3G (search) in 2024 and for paroxysmal nocturnal hemoglobinuria (search) (PNH (search)) later this quarter.
If approved, Omeros will receive a priority review voucher, potentially accelerating FDA review of future drug applications by at least four months.
Omeros Corporation announced that its MASP-3 (search) inhibitor, 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 (search)). This ultra-rare, progressive renal disorder primarily affects children and young adults, often leading to end-stage renal disease within 10 years of diagnosis. There is currently no approved treatment for C3G.
Zaltenibart is designed to inhibit mannan-binding lectin-associated serine protease-3 (MASP-3 (search)), a key activator of the alternative pathway of complement, thereby stopping the conversion of pro-complement factor D (pro-CFD) to mature CFD. By targeting MASP-3, zaltenibart acts as the most proximal inhibitor of the alternative pathway.
Clinical Development Plans
Omeros is advancing zaltenibart for both C3G (search) and paroxysmal nocturnal hemoglobinuria (search) (PNH (search)), an ultra-rare and life-threatening blood disease. The company plans to initiate Phase 3 clinical trials for zaltenibart in C3G in 2024. Phase 3 studies for PNH are slated to begin later this quarter, following productive meetings with regulatory authorities, including an end-of-phase 2 meeting with the FDA and a scientific advice meeting in Europe.
Priority Review Voucher
Companies that receive a rare pediatric disease designation are eligible for a rare pediatric disease priority review voucher from the FDA upon approval of the designated drug for the specified pediatric indication. This voucher allows the recipient to obtain priority review of a New Drug Application (NDA) or Biologics License Application (BLA) for a different product and/or indication, potentially reducing the review time by at least four months. The voucher can be used by the original recipient or sold to another company.
Market Opportunity
The PNH (search) treatment market was valued at $3.9 billion in 2023 and is projected to grow to $10.1 billion by 2032, presenting a significant commercial opportunity for Omeros. Zaltenibart has also received orphan drug designation from the FDA for PNH.
Executive Commentary
"C3G (search) 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. "With zaltenibart clinical studies ongoing in both PNH (search) and C3G and preparations underway to begin Phase 3 trials, we look forward to bringing zaltenibart to market, expanding its list of targeted indications and demonstrating its advantages over other alternative pathway inhibitors."